| Date | Title | Description |
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| Apr 29, 2026 | Insilico's Rentosertib Inhalation Solution Receives IND Clearance for the World's First AI-Driven Candidate to Enter Direct-to-Lung Clinical Study | Following positive Phase IIa results for oral Rentosertib in GENESIS-IPF with good tolerability, a favorable PK profile, and dose-dependent efficacy improvements, the inhalation solution has received IND clearance from the CDE.
The inhalati... |
| 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... |
| Sep 5, 2024 | RARE Navigator Pilot Program Aims to Eliminate Critical Gaps in Support for Patients and Families Newly Diagnosed with a Rare Disease | Global Genes
The rare disease patient advocacy organization Global Genes, has launched a pilot program with Corewell Health Helen DeVos Children's Hospital (HDVCH) in Grand Rapids, Mich., to address gaps in support faced by newly diagnosed ... |
| Aug 22, 2024 | Global Genes' Annual Week in RARE Event Heading to Kansas City | Global Genes
Global Genes is taking Week In RARE on the road to Kansas City, after having hosted this annual event in California for over 12 years. Moving the location annually will help make the event more accessible and equitable to the g... |
| Jul 17, 2024 | Global Genes and STXBP1 Foundation Enter Partnership to Use RARE-X Research and Data Collection Platform for Natural History Study | Global Genes
Global Genes and STXBP1 Foundation are working with Children's Hospital of Philadelphia on a multi-site natural history study, the STARR study, to use the RARE-X research and data collection platform to collect patient-reported... |
| Jun 13, 2024 | Global Gene's RARE-X and Sleep Consortium Launch Sleep Data Collection Initiative to Accelerate Development of Therapies | Global Genes
The Sleep Data Collection Initiative, the first consortium on the RARE-X platform for the collection and sharing of patient-owned data, brings together patient advocacy organizations, researchers, and drug developers looking to... |
| Mar 14, 2024 | Rare Disease Advocates Learn to Accelerate Therapeutic Development at 9th RARE Drug Development Symposium | RARE Drug Development Symposium
The RARE Drug Development Symposium, hosted by Global Genes and the Orphan Disease Center of the University of Pennsylvania, equips advocates with the knowledge, skills and connections they need to advance th... |
| Feb 28, 2024 | Global Genes Heads to Washington D.C. for Rare Disease Day Events | Global Genes
Global Genes CEO Charlene Son Rigby to participate in panel discussion at White House Forum
ALISO VIEJO, Calif., Feb. 28, 2024 /PRNewswire-PRWeb/ -- Charlene Son Rigby, CEO of the rare disease patient advocacy organization Glob... |
| Jul 22, 2023 | Insilico Medicine Nominates Preclinical Candidate of Inhalation Solution for ISM001-055, the First Generative AI IPF Drug | Insilico Medicine merges nebulizer device with AI-driven drug discovery;
Potential advantages for the inhalation solution include a smaller effective dose, larger safety window and reduced side effects;
As demonstrated in preclinical studie... |
| Jul 12, 2023 | A young girl’s custom gene therapy hints at a framework for tailored rare disease treatments | When Timothy Yu developed milasen, a custom drug for a young girl named Mila with Batten disease, he ignited a spark in the field of personalized medicine. Milasen was the first medicine specifically designed for a single person, and it was... |
| Jun 27, 2023 | Rare Disease Patient Advocates Gather in San Diego for a Week of Events to Connect, Learn, and Empower | Week in RARE events "We are creating an unparalleled opportunity for rare disease advocates to engage with each other, build relationships, and help them understand what they can do to change what it means to live with a rare disease d... |
| May 31, 2023 | Global Genes’ RARE-X Issues Open Call to Researchers to Participate in the Inaugural Xcelerate RARE Challenge | We're bringing together researchers and data scientists, many of whom don’t normally work on these rare diseases. Teams will have the opportunity to collaborate and vet analytical approaches and therapeutic strategies. - Karmen Trzupek, Sen... |
| May 23, 2023 | Sleep Consortium Launches Sleep Patient Data Collection Initiative to Accelerate Therapeutic Development | "Sleep Consortium’s new patient data collection platform is poised to become a critical driver for learnings, advancements, and patient-centered progress to improve countless lives.” -- Julie Flygare, President and CEO, Project Sleep.
... |
| Apr 11, 2023 | Can gene editing kill deadly diseases? | Two clinics sit adjacent to one another in a new hospital in a medium-sized city in the developed world.
In one, a family waits for a long-scheduled appointment. Their daughter suffers from a rare inherited disease. One of her genes encodes... |
| Feb 7, 2023 | Global Genes Announces Completion of RARE-X Merger and Strategic Direction for Combined Organization | “The combination of Global Genes and RARE-X provides individuals, families, and advocates with an integrated set of essential services to help them raise support and awareness, accelerate the development of treatments, and power those effor... |
| Jan 17, 2023 | RARE-X to Launch an Open Science Data Challenge to Address Rare Neurodevelopmental Diseases | “This challenge should demonstrate to researchers, drug developers, and patient advocates that people today are already using our data platform to unleash a new understanding of rare diseases to drive improvements in the diagnosis and treat... |
| Sep 6, 2022 | COMBINEDBrain Received the Global Genes 2022 Health Equity RARE Patient Impact Grant | NASHVILLE, TENNESSEE, UNITES STATES, September 6, 2022 /EINPresswire.com/ -- COMBINEDBrain Received the Global Genes 2022 Health Equity RARE Patient Impact Grant
COMBINEDBrain takes steps to break down language barriers perpetuating health ... |
| Apr 29, 2022 | Undiagnosed Patient Organizations Begin Leveraging RARE-X | "we're very fortunate to have partners like RARE-X, who have a similar vision of a scalable, more equitable collaborative future for all healthcare starting with the ultra-rare, starting with the undiagnosed,” said Cristina Might, exec... |
| Feb 9, 2022 | A mother, shaped by tragedy, embarks on a mission to advance custom medicines | For weeks, Julia Vitarello avoided the room in her home. The fairy curtains she sewed. The sheets. The quiet. But one day, she placed her desk by the window. There, she began spending long hours on a single-minded mission: advancing custom ... |
| Oct 21, 2021 | Global Genes and RARE-X Partner to Enable Patient Data Collection for Rare Disease Groups | Global Genes, a leading international rare disease advocacy organization, announced a partnership with RARE-X to equip patient communities to leverage their data to drive rare disease research and drug development.
RARE-X, a technology nonp... |
| Aug 24, 2021 | AllStripes : Announces $50 Million Series B Financing to Advance Global Rare Disease Research | AllStripes (“Company”), a healthcare technology company dedicated to unlocking treatments for people affected by rare diseases, today announced the Company completed a $50 million Series B financing round.
This press release features multim... |
| Aug 5, 2021 | New Guidebook for Caregivers of Children with Rare and/or Serious Illnesses | The Circle of Care Guidebook for Caregivers of Children with Rare and/or Serious Illnesses addresses the ups and downs of caring for a child living with advanced illness and medical complexity, offering new resources, personal stories, and ... |
| 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. |
| Jun 11, 2020 | What being stricken with Covid-19 taught a biotech CEO | On March 14, at 10 a.m., I got the call that I had tested positive for SARS-CoV-2, the virus that causes Covid-19. At first, I was incredulous, trying to understand where I might have contracted it. Next, I got worried. Who had I been in co... |
| Oct 17, 2019 | Pelosi’s drug pricing plan puts medical innovation at risk | The future of medical innovation in America is at a crossroads.
Biopharmaceutical science is accelerating at a record pace, leading to new discoveries that are radically reshaping our ability to fight disease. At the same time, politicians ... |
| Dec 4, 2018 | Government investigation finds flaws in the FDA’s orphan drug program | The GAO uncovered inconsistent and often incomplete reviews early in the process of designating medicines as orphan drugs and recommended “executive action” to fix the system. In some cases, FDA reviewers failed to show they had checked how... |
| Apr 30, 2018 | Delays in adding new newborn screening tests harm babies, families | Individuals with genetic diseases often face an excruciatingly long diagnostic odyssey. Between the time they first experience symptoms and the time they get a definitive diagnosis, some see the disease progress, others suffer irreversible ... |
| Nov 30, 2015 | No longer invisible: The faces of rare disease | A mother who spent years struggling to diagnose her daughter’s illness has found a new tool to draw attention to rare diseases: oil on canvas.
Patricia Weltin, a single mom raising two daughters with rare diseases, described years of frustr... |
| Oct 22, 2014 | Personal Health Record provider CareSync raises $4.25 million for national expansion | It has more than 10,000 users, mainly on the consumer side, who can access their history and other services such as appointments and prescriptions, through a mobile device or laptop. The consumer side also has a wider geographic footprint. ... |
| Jul 2, 2013 | Got an idea to disrupt pediatric healthcare? Boston Children’s wants to hear about it. | It has to be said that when it comes to innovation, there’s loads of interesting developments in pediatrics, from intubation to telemedicine to rare diseases. The only thing is, it’s often overshadowed by the pace of developments for improv... |
| Apr 9, 2013 | Rare disease survey: Physician-patient networks could cut down diagnosis time (infographic) | Among the reasons why it takes so long to diagnose a rare disease are, not surprisingly, they occur so infrequently that a lot of physicians lack experience identifying them and don’t recognize the symptoms. That means patients have to have... |
| Rare disease patients push for access to treatment in Panama | Reporting for this story was supported by a grant from the National Press Foundation.
Panama City, Panama – Maria has lost track of how many times she has seen a healthcare worker over the past three years. But despite innumerable medical c... |
| Rare disease survey: Physician-patient networks could cut down diagnosis time (infographic) | A new report is recommending developing more online networks as a way to shorten the more than seven year timeframe to diagnose a rare condition.
The report by drug developer Shire envisions these networks as connecting specialists with pat... |
| Got an idea to disrupt pediatric healthcare? Boston Children’s wants to hear about it. | It has to be said that when it comes to innovation, there’s loads of interesting developments in pediatrics, from intubation to telemedicine to rare diseases. The only thing is, it’s often overshadowed by the pace of developments for improv... |
| Government investigation finds flaws in the FDA’s orphan drug program | The Food and Drug Administration has failed to ensure that drugs given prized rare-disease status meet the intent of a 35-year-old law, federal officials revealed in a report Friday.
The Government Accountability Office, which spent more th... |