Rare disease news, mapped to the diseases behind the headlines. AI-classified across pipeline, policy, funding, science, and community.
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Regenxbio's RGX-202 gene therapy for Duchenne muscular dystrophy (DMD) demonstrated significant improvements in patient outcomes, exceeding expected disease progression at 12 and 18 months post-treatment in a pivotal trial. The company plans to submit a Biologics License Application (BLA) in mid-2026 via an accelerated approval pathway.
EMA recommended 104 medicines for marketing authorization in 2025, including 38 with new active substances not previously authorized in the EU. This highlights ongoing innovation in the pharmaceutical landscape.
The CDC is seeking public comment on a proposed information collection project titled Poison Center Collaborations for Public Health Emergencies (PCCPHE). This initiative aims to enhance data collection efforts related to public health emergencies, as mandated by the Paperwork Reduction Act.
The Department of Health and Human Services (HHS) has updated the poverty guidelines to reflect last year's price increases as measured by the Consumer Price Index. This update is crucial for determining eligibility for various health and social services programs.
Current discussions highlight the need for temporary treatments for metachromatic leukodystrophy (MLD) before gene therapy can be administered. The focus is on bridging therapies for early juvenile or pre-symptomatic children to manage symptoms effectively.
The HHS is undergoing a review under the Paperwork Reduction Act, which may impact data collection processes. Stakeholders should monitor potential changes that could affect reporting requirements.
The CDC is seeking public comment on a proposed data collection project titled Maritime Activity Illness and Death Reporting. This initiative aims to enhance the utility of government information while reducing public burden, as mandated by the Paperwork Reduction Act.
The CDC announces a public meeting of the Advisory Board on Radiation and Worker Health, allowing for written comments but no public comment period. This meeting aims to discuss issues related to radiation exposure and worker health.
A new Salmonella outbreak has been linked to Live It Up Super Greens supplement powders, affecting 45 individuals across 21 states. The CDC is investigating the source of the contamination to prevent further cases.
The U.S. biotech sector is expected to rebound by 2026, with increased IPO activity as investor confidence grows amid a favorable interest rate environment. Experts suggest that companies with mid-to-late-stage drug development will attract significant interest.
Washington University in St. Louis will host its annual Rare Disease Day Symposium to raise awareness and foster collaboration among researchers and clinicians in the field of rare diseases. This event aims to inspire the next generation of scientists and healthcare professionals to engage in rare disease research.
EMA and FDA have established ten guiding principles for the use of artificial intelligence in drug development, marking a significant step in EU-US collaboration on medical technologies. This initiative aims to enhance the integration of AI throughout the medicines lifecycle, reflecting its growing importance in the industry.
Virginie Hivert, Acting CEO of EURORDIS, shares insights on recent developments within the organization, emphasizing transparency and engagement with members. This communication aims to strengthen the community's connection and awareness of ongoing efforts in rare disease advocacy.
The U.S. Department of Health and Human Services is hosting a virtual Tribal consultation regarding proposed modifications to the HIPAA Privacy Rule aimed at enhancing coordinated care and individual engagement. This initiative seeks to address barriers within the current privacy framework.
The Federal Register has issued a notice regarding a hearing opportunity to assess Minnesota's compliance with Medicaid program integrity provisions. This review focuses on adherence to Title XIX of the Social Security Act, addressing concerns related to fraud, waste, and abuse.
FDA grants Breakthrough Therapy Designation to Ipsen’s IPN60340 (ICT01) for first-line treatment of unfit acute myeloid leukemia. This designation aims to expedite the development and review process for this promising therapy.
Experts highlight the urgent need for innovative funding models in rare disease therapies, calling it a 'modern-day civil rights issue.' The discussion emphasizes the collaboration between venture capital, patient advocacy, and research to ensure access to cures for all, regardless of commercial viability.
The CDC is seeking public comment on the proposed revision of the Maternal Mortality Review Information Application (MMRIA) to enhance data collection efforts. This initiative aims to reduce public burden while improving the utility of government information related to maternal mortality.
The CDC is seeking public comment on a proposed information collection project for the National Disease Surveillance Program. This initiative aims to enhance data collection efforts while minimizing public burden, as mandated by the Paperwork Reduction Act.
The Federal Register has updated the Master List of items potentially subject to face-to-face encounter and prior authorization requirements, impacting HCPCS codes. These changes may affect reimbursement processes for various healthcare items.
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