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| Jun 17, 2026 | Final rules for Medicaid work requirements are out. Here’s what you need to know | Getting your Trinity Audio player ready...
By Sam Whitehead, KFF Health News
The Trump administration has issued final rules on how states should ensure that millions of Medicaid enrollees prove they’re working or completing other activitie... |
| Jun 14, 2026 | Menarini Group Reports Data from the Phase 3 SENTRY Trial of Selinexor Plus Ruxolitinib in Myelofibrosis at The European Hematology Association (EHA) 2026 Congress | The combination of selinexor plus ruxolitinib met the first co-primary endpoint demonstrating a statistically significant improvement of spleen volume reduction (SVR35) of 49.8% for the combination arm vs 28% for the control arm at week 24.... |
| Mar 10, 2026 | FDA approves leucovorin as first drug for rare genetic disorder, after touting it as autism treatment | The Food and Drug Administration approved a decades-old prescription vitamin called leucovorin as the first treatment for a rare genetic disorder in certain adults and children.
The move comes months after the Trump administration touted le... |
| Mar 2, 2026 | ‘Beast Games’ winner Jeff Allen hopes finding a cure for his son’s rare disease will unlock new treatments for other brain conditions | To share your story, or find out how to get involved with CNBC Cures, email [email protected]
Jeff Allen’s son Lucas was diagnosed in 2019 with creatine transporter deficiency, a gene mutation that deprives the brain and muscles of essent... |
| Jan 8, 2026 | CNBC launches CNBC Cures to help 30 million Americans with rare diseases | If you’d like to share your story, or receive more information, email us: [email protected]
CNBC Cures aims to help raise awareness of rare diseases and improve patient outcomes for people living with them.
Led by “Squawk Box” anc... |
| May 6, 2025 | Calquence combination approved in EU for 1L MCL | Calquence combination approved in EU for 1L MCL
Tue, May 06, 2025 08:02 CET Report this content
06 May 2025
Calquence plus chemoimmunotherapy approved in the EU
as first and only BTK inhibitor for 1st-line mantle cell lymphoma
Approval base... |
| Mar 31, 2025 | AstraZeneca's Breakthroughs: A New Dawn in Cancer Treatment | In the realm of oncology, AstraZeneca is carving a path through the dense forest of cancer treatment. Two recent approvals highlight this journey: Calquence for mantle cell lymphoma (MCL) and Imfinzi for muscle-invasive bladder cancer (MIBC... |
| Mar 31, 2025 | Calquence recommended for EU approval in 1L MCL | Calquence recommended for EU approval in 1L MCL
Mon, Mar 31, 2025 08:05 CET Report this content
31 March 2025
Calquence plus chemoimmunotherapy recommended for approval in the EU by CHMP as first and only BTK inhibitor for 1st-line mantle c... |
| Feb 21, 2025 | FIRST AND ONLY TREATMENT FOR CHRONIC HYPOPARATHYROIDISM APPROVED IN AUSTRALIA | YORVIPATH® (palopegteriparatide) is now registered by the Therapeutic Goods Administration (TGA) for the treatment of chronic hypoparathyroidism in adults1
First-in-class novel PTH replacement therapy
Australia becomes the first country to ... |
| Jul 23, 2024 | NORD Report Highlights Benefit of Pediatric Drug Development Incentive Program for Children Living with Rare Diseases | Effective Rare Pediatric Disease Priority Review Voucher (RPD PRV) Program Set to Expire September 30th without Congressional Action
WASHINGTON, July 23, 2024 /PRNewswire/ -- Today, the National Organization for Rare Disorders (NORD) releas... |
| Jun 15, 2024 | His son was born with a rare condition, so this father donated a kidney. He "always knew it was going to be him." | When Stephen Munari's son Jaren was born, Munari knew he would one day donate an organ to help him.
The day before his birth, doctors told Jaren's parents that their son would have just the one kidney, which was "on the small side,&quo... |
| Jun 11, 2024 | Japanese Researchers Discover Treating Skin Diseases Possible Through Healthy Skin Transplants | By Joseph Henry
Published Jun 11, 2024 11:53AM EDT
Share on Facebook Share on Twitter Share on LinkedIn Share on Reddit Share on Flipboard Share on Pocket
If you think that your skin disorder is incurable even though you have consumed all y... |
| May 2, 2024 | Calquence combination improved PFS in 1L MCL | Calquence combination improved PFS in 1L MCL
Thu, May 02, 2024 08:00 CET Report this content
02 May 2024
Calquence combination regimen demonstrated statistically significant and clinically meaningful improvement in progression-free survival... |
| May 1, 2024 | National Organization for Rare Disorders (NORD) Names Pamela K. Gavin as CEO | Peter L. Saltonstall Retires and Will Serve in Consultative Role
QUINCY, Mass., May 1, 2024 /PRNewswire/ -- The National Organization for Rare Disorders (NORD) announced today that Pamela K. Gavin has been named CEO, effective immediately. ... |
| Apr 29, 2024 | Newborn Screening Emergency: Urgent Call for Action to Identify 10,000+ Undiagnosed Rare Diseases | RARE PRS #12
The push for a transformative U.S.-India partnership in newborn screening (NBS) sets the stage for a seismic shift in tackling rare diseases, spotlighting the need to prepare for the digital AI revolution in global healthcare. ... |
| Mar 14, 2024 | AstraZeneca Acquisition Brings Drug to Fill a Rare Disease Gap Left by Takeda | AstraZeneca is bolstering its rare disease pipeline with a deal to buy Amolyt Pharma, a biotech company in late-stage development with a therapy designed to treat a rare hormone deficiency whose only FDA-approved treatment will soon cease p... |
| Feb 26, 2024 | RAISING AWARENESS FOR RARE DISEASE DAY: FOCUS ON HUNTER SYNDROME | Project Alive VP, Kyle Underwood goes to Washington, DC to lobby for support for rare diseases day. LOS ANGELES, CALIFORNIA, UNITED STATES, February 26, 2024 /EINPresswire.com/ -- Project Alive a leading advocate for rare diseases, proudly ... |
| Jan 16, 2024 | Vertex’s CRISPR Gene Therapy Lands Another FDA Nod in a Rare Blood Disease | A one-time CRISPR-based gene therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics now has an additional FDA approval as a treatment for the rare blood disease beta thalassemia.
The affirmative decision announced Tuesday for t... |
| Dec 6, 2023 | Novartis Nabs FDA Nod for Rare Disease Drug Rival to AstraZeneca Meds | A rare blood disorder treatable with infused medications now has its first oral drug alternative. FDA approval of the new drug from Novartis positions the pill to compete directly against two blockbuster AstraZeneca products.
The rare disea... |
| Dec 4, 2023 | Gexval Co., Ltd. We have implemented a capital increase through third-party allocation. | Gexval Co., Ltd.
We implemented a third-party allotment capital increase.
~Toward accelerating development and adding pipeline additions in Australia~ ……
Gexval Co., Ltd. (President and CEO: Juran Kato, location: Fujisawa City, Kanagawa Pre... |
| Dec 4, 2023 | Gexval Co., Ltd. We have implemented a capital increase through third-party allocation. | 0 0 votes
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| Nov 13, 2023 | Ajinomoto Adds Gene Therapy to CDMO Scope via $554M Forge Bio Buyout | Ajinomoto’s presence in contract pharmaceutical manufacturing spans small molecules, antibody drug conjugates, and nucleic acid-based therapies. The Japanese company is adding gene therapy to the list with the acquisition of Forge Biologics... |
| Nov 3, 2023 | State Prescription Drug Affordability Boards could end up limiting access to the life-saving cystic fibrosis medication Trikafta | I was living on the precipice of end-stage disease when I enrolled in the clinical trial that resulted in the historically fast Food and Drug Administration approval for the game-changing cystic fibrosis drug Trikafta. Five years later, the... |
| Oct 5, 2023 | Kyowa Kirin Buys Into Gene Therapy With Orchard Therapeutics Acquisition | Kyowa Kirin is expanding its scope to genetic medicines by acquiring Orchard Therapeutics, a company whose gene therapy for a rare inherited metabolic disorder is commercialized in Europe and is on track for an FDA decision in the first qua... |
| Oct 5, 2023 | I'm planning my baby's 1st birthday party while thinking about her funeral | Parenting I'm planning my baby's 1st birthday party while thinking about her funeral
As told to Kelly Burch
2023-10-05T12:04:01Z
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| Sep 26, 2023 | Health | This lab’s gene test can find more rare diseases. This is what it can do for the ‘diagnostic odyssey’ of families. | There are more than 7,000 rare disorders, and many cannot be diagnosed in a doctor’s office.
That’s why genetic sequencing is so important.
Now The Jackson Laboratory in Farmington is able to sequence the entire genome, an advance that will... |
| Jul 13, 2023 | What is Bell's palsy? What to know after Tiffany Chen's diagnosis reveal | The cause is a mystery — and the symptoms can be distressing. Bell's palsy comes on without warning, bringing partial paralysis or weakness to a person's face.
The condition affects thousands of people in the U.S. every year. And now Tiffan... |
| Jun 13, 2023 | Ipsen Itching Drug Scratches Off a Second FDA Approval in Rare Liver Indication | An Ipsen drug developed to treat the severe itching that accompanies certain liver disorders is now FDA approved for treating this complication in patients with Alagille syndrome. It’s the second approved indication for Bylvay, a drug that ... |
| May 13, 2023 | No rodent will be CHASING these huge blocks of cheese | The winner of the 2019 cheese rolling competition, He rolled, fell, and cartwheeled his way to victory. Image:traavelbeginsat40.com
Cheese and rolling a wheel of it down a very, very steep hill, just outside the city of Gloucester, England ... |
| May 10, 2023 | Rare Disease Drug Approval Brings New Competition to Meds from Sanofi, Takeda | A drug developed by partners Protalix BioTherapeutics and Chiesi Group has won FDA approval, a regulatory decision that follows the product’s European approval last week, introducing new competition for the treatment of a rare disorder with... |
| Apr 11, 2023 | Chinook Therapeutics Pauses Kidney Disease Drug Trial Following Adverse Event Report | Chinook Therapeutics has stopped testing of a liver disease therapy after a healthy patient in an early-stage study developed an unexpected complication believed to be a reaction to the experimental drug.
The serious adverse event prompted ... |
| Feb 23, 2023 | FDA Continues Important Work to Advance Medical Products for Patients with Rare Diseases | Image
By: Robert M. Califf, M.D., Commissioner of Food and Drugs and Sandy Retzky, D.O., J.D., M.P.H., Director, Office of Orphan Products Development
Patients with rare diseases are experts in their health condition. They provide a unique ... |
| Feb 21, 2023 | Blood Clotting Biotech Hemab Hauls In $135M to Drug Rare Bleeding Disorders | Clotting disorders can be treated by infusing patients with clotting proteins they lack, or with drugs that replace the function of these proteins. Gene therapies may soon become another option. Hemophilia A and B are perhaps the best-known... |
| Dec 22, 2022 | Atara Approval Marks Cell Therapy First; Entrada on Hold, Bluebird Freed & More | When patients undergo transplant procedures, the immunosuppressive drug regimen that prevents rejection of the organ or stem cells can lead to a potentially deadly complication. This week, the European Commission approved a cell therapy to ... |
| Nov 28, 2022 | Genome writing startup Replay receives a $1.5m grant from Bill Gates for its immune silencing technology to bring “big DNA” treatments to diseases | One in ten Americans suffers from a rare disease, according to the National Institutes of Health (NIH). The agency found that there are approximately 7,000 rare diseases affecting between 25 and 30 million Americans. That’s why San Diego-ba... |
| Nov 17, 2022 | Woman with a rare brain disease says doctors repeatedly dismissed her condition, blaming it on mental illness | Simpson in the hospital after being admitted with stroke-like symptoms in January 2021. Courtesy of Daisy Simpson
Daisy Simpson, 34, says her brain disease was overlooked due to an earlier psychosis diagnosis.
Simpson says she believes the ... |
| Nov 1, 2022 | Karyopharm and Menarini Group Announce Orphan Medicinal Product Designation from the European Commission for Selinexor for the Treatment of Myelofibrosis | NEWTON, Mass. and FLORENCE, Italy, Nov. 1, 2022 /PRNewswire/ -- Karyopharm Therapeutics Inc. (NASDAQ: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, and the Menarini Group ("Menarini"), a priva... |
| Oct 24, 2022 | Meeting the needs of rare disease patients: Improved access to specialty pharmaceuticals, gene and cell therapies | Anyone who steps into the shoes of a person with a rare disease will experience not only the disheartening prospects of deteriorating health but also the pressure to arrange for effective treatment sooner and prevent morbidity and mortality... |
| Oct 13, 2022 | RDDC and NORD Announce New Partnership to Help Identify Barriers for Rare Disease Patients and their Caregivers | New initiative seeks to identify gaps experienced by patients and caregivers in underrepresented communities along their rare disease diagnosis and treatment journey
WASHINGTON, Oct. 13, 2022 /PRNewswire/ -- Today, the Rare Disease Diversit... |
| Sep 21, 2022 | Artist finds private medical record photos in popular AI training data set | Enlarge / Censored medical images found in the LAION-5B data set used to train AI. The black bars and distortion have been added.
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| Sep 20, 2022 | Apple wins a Patent for a Mixed Reality HMD that could assist those with various stages of Alzheimer's Disease
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Search | Today the U.S. Patent and Trademark Office officially granted Apple a patent that relates to head-mounted devices, and, more particularly, to head-mounted devices that can detect and respond to a user's behavior. More specifically, Apple's ... |
| Sep 16, 2022 | Another Intellia CRISPR therapy flashes potential for one-time, in vivo treatment | An experimental CRISPR-based treatment from Intellia Therapeutics now has encouraging early clinical data showing it led to reductions in levels of a liver protein that drives a rare and potentially fatal swelling disorder. The results repo... |
| Aug 25, 2022 | Ultomiris approved in Japan for gMG | Ultomiris approved in Japan for gMG
Thu, Aug 25, 2022 08:10 CET Report this content
25 August 2022 07:10 BST
Ultomiris approved in Japan for the treatment of adults with generalised myasthenia gravis
First and only long-acting C5 complement... |
| Aug 9, 2022 | Ashton Kutcher lost his sight, ability to walk, and hearing for close to a year due to rare autoimmune disease | Entertainment Ashton Kutcher lost his sight, ability to walk, and hearing for close to a year due to rare autoimmune disease
Azmi Haroun and Allana Akhtar
2022-08-08T21:03:08Z
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| Jul 25, 2022 | Ultomiris recommended for approval in the EU by CHMP for the treatment of adults with generalised myasthenia gravis | Ultomiris recommended for approval in the EU by CHMP for the treatment of adults with generalised myasthenia gravis
Mon, Jul 25, 2022 08:06 CET Report this content
First and only long-acting C5 complement inhibitor showed early effect and d... |
| Jul 25, 2022 | Ultomiris recommended for EU approval for gMG | Ultomiris recommended for EU approval for gMG
Mon, Jul 25, 2022 08:05 CET Report this content
25 July 07:05 BST
Ultomiris recommended for approval in the EU by CHMP for the treatment of adults with generalised myasthenia gravis
First and on... |
| Jun 13, 2022 | UPSC Key-June 13, 2022: Why and What to know about ‘Article 80 of the Constitution’ to ‘Ramsay Hunt Syndrome’ for UPSC CSE? | Important topics and their relevance in UPSC CSE exam for June 13, 2022. If you missed the June 10, 2022 UPSC key from the Indian Express, read it here
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14 of 15 states had asked for higher MSP for paddy than what Centre announced... |
| May 26, 2022 | CONSILIENT HEALTH LAUNCHES GHRYVELIN™ (macimorelin), A NOVEL ORAL DIAGNOSTIC TEST FOR ADULT GROWTH HORMONE DEFICIENCY | FOR UK/EUROPEAN MEDICAL AND TRADE MEDIA ONLY
DUBLIN, IRELAND, May 26, 2022 /EINPresswire.com/ -- Consilient Health has today announced the launch of Ghryvelin™ (macimorelin), the first oral test licensed for diagnosing Growth Hormone Defici... |
| May 26, 2022 | CONSILIENT HEALTH LAUNCHES GHRYVELIN™ (macimorelin), A NOVEL ORAL DIAGNOSTIC TEST FOR ADULT GROWTH HORMONE DEFICIENCY | FOR UK/EUROPEAN MEDICAL AND TRADE MEDIA ONLY DUBLIN, IRELAND, May 26, 2022 /EINPresswire.com/ -- Consilient Health has today announced the launch of Ghryvelin™ (macimorelin), the first oral test licensed for diagnosing Growth Hormone Defici... |
| Apr 28, 2022 | Ultomiris approved in the US for adults with gMG | Ultomiris approved in the US for adults with gMG
Thu, Apr 28, 2022 08:00 CET Report this content
28 April 2022 07:00 BST
Ultomiris approved in the US for adults with generalised myasthenia gravis
First and only long-acting C5 complement inh... |
| Feb 16, 2022 | Backed by $100M, Star Therapeutics sets out to find constellations of rare disease | Rare disease offers plenty of drug research opportunities simply because there are so many diseases yet so few medicines. For the more than 7,000 rare diseases that have been identified, there are only about 650 FDA-approved therapies. Star... |
| Feb 14, 2022 | Management of Pediatric Clinical Trials Calls for Urgent Technological Innovation | Of the 93% of the overall American population affected by rare diseases, 50% are children. Meanwhile, the time it takes for a patient to get an accurate rare disease diagnosis is six to eight years. A consultative and collaborative human-ce... |
| Feb 14, 2022 | Precision medicine startup Variantyx lands $41.5M in funding to treat rare diseases using whole-genome sequencing | One in ten Americans suffers from a rare disease, according to the National Institutes of Health (NIH). The agency found that there are approximately 7,000 rare diseases affecting between 25 and 30 million Americans. That’s why Framingham, ... |
| Feb 11, 2022 | Outlook dims for ProQR’s RNA therapy for vision loss following failure in key test | An RNA therapy from ProQR Therapeutics has failed a pivotal test in an inherited eye disorder, an unexpected setback to the biotech company’s most advanced program. According to the preliminary results, patients who received the experimenta... |
| Feb 8, 2022 | GenomeUP chiude un seed round da €1,1m | GenomeUp, startup italiana attiva nel settore digital biotech, ha sviluppato JuliaOmix™: un ecosistema di servizi digitali dotati di IA per il supporto alla diagnosi delle malattie genetiche e per la digitalizzazione dei processi di laborat... |
| Jan 24, 2022 | Pfizer and OPKO Provide Update on the Biologics License Application for Somatrogon for Pediatric Growth Hormone Deficiency | NEW YORK & MIAMI-Pfizer Inc. (NYSE: PFE) and OPKO Health, Inc. (NASDAQ: OPK) announced that the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) for the Biologics License Application (BLA) for somatrogon. ... |
| Jan 21, 2022 | Japan's MHLW Approves Pfizer and OPKO's NGENLA (Somatrogon), a New Long-Acting Treatment for Pediatric Growth Hormone Deficiency | TOKYO and MIAMI - Pfizer Japan Inc. and OPKO Health, Inc. (NASDAQ: OPK) announced that the next generation long-acting growth hormone injection, NGENLA (somatrogon) Inj. 24 mg Pens and 60 mg Pens, has been approved by the Ministry of Health... |
| Jan 20, 2022 | Japan's MHLW Approves Pfizer and OPKO's NGENLA® (Somatrogon), a New Long-Acting Treatment for Pediatric Growth Hormone Deficiency | TOKYO and MIAMI, Jan. 20, 2022 (GLOBE NEWSWIRE) -- Pfizer Japan Inc. and OPKO Health, Inc. (NASDAQ: OPK) announced today that the next generation long-acting growth hormone injection, NGENLA® (somatrogon) Inj. 24 mg Pens and 60 mg Pens, has... |
| Jan 7, 2022 | Amylyx Pharma’s IPO lands $190M as ALS drug begins FDA review | Amylyx Pharmaceuticals has joined the public markets with a $190 million IPO that will support the company as it shepherds its amyotrophic lateral sclerosis drug through regulatory review, and if all goes well, a commercial launch as a new ... |
| Dec 31, 2021 | My son will be fine, she told the doctors, because he is the Lord God Jesus Christ | Editor’s note: This essay, initially published a year before the emergence of the global coronavirus pandemic, takes on new meaning in light of the failure to find common language around Covid-19.
Medical parables are day-to-day stories pas... |
| Dec 29, 2021 | FDA sets decision date for Amylyx Pharma ALS drug that improves neuron survival | Neurons die. It’s a hallmark of amyotrophic lateral sclerosis and other neurodegenerative disorders, but drug developers have been mostly stymied in their attempts to help these nerve cells survive. Amylyx Pharmaceuticals designed its exper... |
| Dec 27, 2021 | Heart drug’s trial failure busts bid to best Pfizer, but BridgeBio still bets on coming data | A BridgeBio Pharma drug that the company had touted as potentially better than an approved Pfizer product at addressing the protein at the root of a rare heart disorder has instead failed to beat a placebo in the first part of a pivotal cli... |
| Dec 21, 2021 | Ultomiris accepted for FDA Priority Review for gMG | Ultomiris accepted for FDA Priority Review for gMG Tue, Dec 21, 2021 08:00 CET
21 December 2021 07:00 GMT
Ultomiris regulatory submission accepted under FDA Priority Review in the US for adults with generalised myasthenia gravis
Submission ... |
| Nov 30, 2021 | After Phase 3 failure busts blockbuster bid, Deciphera restructures; staff slashed 35% | Deciphera Pharmaceuticals is stopping work on two programs, focusing its research efforts on two others, and cutting its headcount by 35% in a corporate restructuring. The moves announced Tuesday come weeks after clinical trial data hoped t... |
| Nov 21, 2021 | BioMarin Pharma sees big things for first FDA-approved dwarfism drug | Children whose dwarfism stems from achondroplasia have had few treatment choices other than surgeries to address spinal or limb complications. A drug is now available that could head off some of those problems in the first place. The FDA ha... |
| Nov 20, 2021 | 8 reasons why your hands might be tingling and how to treat it | Reviews Chevron icon It indicates an expandable section or menu, or sometimes previous / next navigation options.
Health 8 reasons why your hands might be tingling and how to treat it
Written by Kelly Burch; edited by Jessica Orwig
2021-11-... |
| Nov 15, 2021 | Horizon Therapeutics plc Receives CHMP Positive Opinion for UPLIZNA (inebilizumab) as a Monotherapy for the Treatment of Adult Patients with Neuromyelitis Optica Spectrum Disorder (NMOSD) | DUBLIN - Horizon Therapeutics plc (Nasdaq: HZNP) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending marketing authorization for UPLIZNA... |
| Nov 12, 2021 | Advanced diagnosis for patients with rare conditions | This week, results from the 100,000 Genomes Project showed that whole genome sequencing improves the diagnosis of rare diseases and shortens diagnostic journeys for patients.
Dr Antony Smith, who leads LifeArc’s assay development and diagn... |
| Nov 10, 2021 | Prevent Blindness Declares Second Annual Thyroid Eye Disease (TED) Awareness Week to Help Educate the Public on Risk Factors, Symptoms, and Resources for Care | Prevent Blindness has declared Nov. 14-20, 2021, as the 2nd Annual Thyroid Eye Disease Awareness Week, to help educate the public on symptoms, treatment options, and more. “As with so many eye diseases and conditions, the earlier TED is dia... |
| Nov 4, 2021 | Cell therapy biotech PlateletBio reels in $75M as it looks ahead to first clinical test | PlateletBio, a company developing a new class of cell therapies based on the biology of platelets, has raised $75.5 million to advance its drug pipeline, including a lead candidate for a rare bleeding disorder on track to reach the clinic n... |
| Oct 29, 2021 | Only a pawn in their game: Orphan drug tax credit on the chopping block again under Dem spending bill | Buried about 1,600 pages into the version of the Build Back Better Act released yesterday afternoon is a pronounced tweak to the orphan drug tax credit that would sharply curtail which clinical testing expenses qualify fo... |
| Oct 28, 2021 | 4 common causes of night sweats and how to get rid of them | Night sweats often occur in menopause. Shutterstock
Night sweats can be caused by hormonal fluctuations or disorders, infection, fever, and more.
Many women will experience night sweats during menopause or perimenopause.
Occasional night sw... |
| Oct 27, 2021 | Public-private partnership launched to accelerate gene therapies for rare diseases | The National Institutes of Health and U.S. Food and Drug Administration unveiled a public-private partnership Wednesday aimed at accelerating gene therapies for the roughly 30 million Americans living with a rare genetic disease.
“They’ve w... |
| Sep 14, 2021 | Aduhelm backlash threatens to reverse progress in FDA’s reviews of rare and ultra-rare disease drugs | The FDA’s approval of Aduhelm to treat Alzheimer’s disease has unleashed criticism about the decision and calls for reform within the agency. As advocates for people living with Barth syndrome — an ultra-rare, life-threatening genetic condi... |
| Sep 2, 2021 | Blood disorder biotech Disc Medicine gets $90M to bring two drugs into Phase 2 | The rare diseases called erythropoietic porphyrias (EP) show up in the blood, but patients feel them most acutely on the skin. Light sensitivity is a major symptom of these enzyme deficiencies affecting red blood cells, and sunlight can cau... |
| Aug 13, 2021 | 5 uses of prednisone, a steroid that reduces inflammation | Long-term use of prednisone may cause fatigue, headaches, diabetes, weight gain, and weakened bones. AP Photo/Robert F. Bukaty
Prednisone is used for conditions like arthritis, blood cell disorders, lupus, and more.
It can also treat inflam... |
| Jul 30, 2021 | BeiGene : Announces Approval in Canada of BRUKINSA® (Zanubrutinib) for the Treatment of Patients with Mantle Cell Lymphoma (Form 8-K) | BeiGene Announces Approval in Canada of BRUKINSA® (Zanubrutinib) for the Treatment of Patients with Mantle Cell Lymphoma
Second approval for BTK Inhibitor BRUKINSA in Canada
CAMBRIDGE, Mass. and BEIJING, China, July 26, 2021 -- BeiGene, Ltd... |
| Jul 28, 2021 | AI-driven Deep Genomics gets $180M to turn biology into informational medicines | Drug discovery and development is advancing by leaps and bounds as genetic medicines progress under a growing number of biotech companies. In the view of Brendan Frey, founder and CEO of startup Deep Genomics, while the drugs of the 20th ce... |
| Jul 26, 2021 | 21 states give rare disease patients a seat at the table. The other 29 need to follow suit | A powerful movement is taking shape in the U.S. rare disease community that could transform the lives of millions of people. That’s right — millions. Even though a single rare disease may affect only a few individuals, there are several tho... |
| Jul 21, 2021 | A researcher in China has died from the rare ‘Monkey B’ virus after dissecting 2 primates | The Monkey B virus can be transmitted from primate to primate. But China has reported its first documented death from the virus – a 53-year-old Beijing-based primate researcher. Koichi Kamoshida/Getty Images
A Beijing-based primate research... |
| Jul 21, 2021 | A researcher in China has died from the rare 'Monkey B' virus after dissecting 2 primates | The Monkey B virus can be transmitted from primate to primate. But China has reported its first documented death from the virus - a 53-year-old Beijing-based primate researcher.
Koichi Kamoshida/Getty Images
A Beijing-based primate research... |
| Jul 15, 2021 | NORD Named an Official Charity Partner of the 2021 TCS New York City Marathon | |
| Jul 7, 2021 | Aduhelm’s accelerated approval offers a promising roadmap for rare neurological diseases | The FDA’s decision to grant accelerated approval to Biogen’s aducanumab (Aduhelm) for the treatment of Alzheimer’s disease was a difficult and bold one that people with the disease, their families, and other drug developers should be applau... |
| Jun 16, 2021 | First Look Showcases Life Science Startups from SoCal Academies | The featured startups span industries from therapeutics to software to green tech. One wants to change the way you experience audio. Another uses CRISPR technology to find a cure for a rare genetic disease. A third wants to repurpose waste ... |
| Jun 8, 2021 | REATA PHARMACEUTICALS, INC.
Reata Pharmaceuticals : 2021 ERA-EDTA Presentation | Master title style
Safety of Bardoxolone Methyl in Pediatric
Patients with Alport Syndrome in
CARDINAL Phase 3 Trial
Bradley Warady, M.D.
Pediatric Nephrology,
Children's Mercy Kansas City,
Kansas City, MO, USA
Authors and Disclosures
Bradl... |
| Jun 2, 2021 | Newer methods may boost gene therapy’s use for more diseases | More On: genetics
Get personalized health insights with this DNA test kit, now 54% off Scientists finally finish decoding entire human genome Dodo DNA discovery could lead to revival of extinct bird How a skin graft could be the next breakt... |
| Jun 2, 2021 | Newer methods may boost gene therapy’s use for more diseases | More On: genetics
Woman sues for $4.8M after DNA test reveals baby swap 20 years ago Local citizen scientists map genetics of Darwin’s Galapagos Family seeks answers after baby suffers broken bones when dog falls on him Discover your dog’s ... |
| May 31, 2021 | Centessa Pharma’s IPO brings in $330M for its new model of drug R&D | Centessa Pharmaceuticals, a young company with a goal of reinventing the traditional business model of drug research and development, has raised $330 million from its IPO to carry out its strategy.
Cheshire, U.K.-based Centessa initially pl... |
| May 26, 2021 | FDA places clinical hold on Larimar’s rare disease drug after deaths in monkey study | In dose-ranging studies for Larimar Therapeutics’ experimental treatment for the rare neuromuscular disorder Friedreich’s ataxia, no problems were observed in patients other than mild to moderate reactions at the injection site. But an undi... |
| May 24, 2021 | My Muscles Spasm Uncontrollably. This Is How I Have Sex | This Is How We Do It
A series about sex and stigma.
See More →
Dystonia, a rare movement disorder that causes uncontrollable muscle spasms like a sudden jerk of the head or contortion of the arm, can be incredibly vexing and unpredictable. ... |
| May 18, 2021 | National Organization for Rare Disorders (NORD) Announces Kay Holcombe as New Board Chair and Dennis Jackman as New Member of its Board of Directors | |
| May 17, 2021 | Nationwide Law Firm Brings Awareness to Necrotizing Enterocolitis | Infants who receive greater than 50% of mother’s own milk (MOM) in the 2 weeks after birth have a significantly decreased risk of NEC.
HOUSTON (PRWEB) May 17, 2021
World NEC Awareness Day is May 17th. NEC is a dangerous disease that primari... |
| Mar 29, 2021 | Inquiry finds no evidence uniQure gene therapy caused cancer in clinical trial | Last December, the FDA placed the clinical hold on the gene therapy, etranacogene dezaparvovec, after a patient who received the treatment two years earlier was diagnosed with a form of liver cancer. Cancer is a known risk of gene therapy, ... |
| Mar 24, 2021 | ProQR’s RNA therapy on track to pivotal tests in rare, inherited form of vision loss | Netherlands-based ProQR is developing its experimental therapy, QR-421a, as a treatment for Usher syndrome. The rare disorder is caused by mutations to genes that provide the instructions for proteins key to hearing, vision, and balance. Th... |
| Mar 15, 2021 | New Zolgensma data demonstrate age-appropriate development when used early, real-world benefit in older children and durability 5+ years post-treatment | Children with SMA treated presymptomatically achieved age-appropriate motor milestones including sitting, standing and walking; required no ventilatory or feeding tube support; and had no serious, treatment-related adverse events
Real-world... |
| Mar 9, 2021 | How Covid-19 is changing rare diseases research | Twenty years after researchers published their first discoveries associated with human genome sequencing, policymakers are fighting to overcome obstacles that hobble the potential of genetic medicine to recognize, diagnose and cure rare dis... |
| Mar 4, 2021 | New Report Finds Medical Treatments for Rare Diseases Account for Only 11% of US Drug Spending; Nearly 80% of Orphan Products Treat Rare Diseases Exclusively | |
| Feb 27, 2021 | 7 science-backed benefits of vitamin B6 and how much you need each day | Spinach is a good source of vitamin B6. Bartosz Luczak / Getty Images
Vitamin B6 is a key nutrient that benefits your immune system, brain, and heart health.
It’s also essential for your body’s metabolism, helping convert food into energy.
... |
| Feb 26, 2021 | Breaking Health Care's Four-Minute Mile | Co-Founder & CEO at Emedgene — on a mission to unlock genomic insights for the world’s health organizations with data-driven AI solutions. |