Rare disease news, mapped to the diseases behind the headlines. AI-classified across pipeline, policy, funding, science, and community.
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Sarepta Therapeutics reports that its gene therapy Elevidys for Duchenne muscular dystrophy shows continued benefits three years post-treatment, despite facing regulatory challenges and a significant workforce reduction. The therapy's efficacy comes amid scrutiny following patient deaths linked to liver injury, highlighting the risks associated with AAV therapies.
The Connect CACNA1C Global Network Conference highlights advancements in understanding CACNA1C-related disorders. This event fosters collaboration among researchers and advocates to improve patient outcomes.
EURORDIS highlights ADOA-plus Day, focusing on awareness and advocacy for individuals affected by ADOA-plus syndrome. This initiative aims to strengthen community support and promote understanding of the disease.
The Muscular Dystrophy Association (MDA) is celebrating Rare Disease Day on February 28 by sharing community stories that highlight advancements in research, care, and advocacy for individuals with rare neuromuscular diseases. This initiative aims to enhance awareness and engagement among those affected by muscular dystrophy, ALS, and related conditions.
The CDC's National Healthcare Safety Network continues to be the leading system for tracking healthcare-associated infections in the U.S. This update highlights its critical role in improving patient safety and infection control practices.
The CDC highlights work-related risks for heart disease and outlines prevention strategies. This initiative aims to raise awareness and promote workplace health policies to mitigate these risks.
The CDC has released new resources for vector control professionals, including guidelines and best practices for mosquito control. These materials aim to enhance public health efforts in managing mosquito-borne diseases.
The CDC emphasizes the importance of keeping children up-to-date on vaccinations to protect against preventable diseases. This guide serves as a resource for parents and caregivers to navigate vaccination schedules.
The U.S. Department of Health and Human Services rescinded guidance on nondiscrimination protections under the Church Amendments, aligning with Executive Orders aimed at enforcing the Hyde Amendment. This change may impact funding and regulatory practices related to abortion services.
The AHRQ has delisted MedExpress as a patient safety organization due to non-compliance with the Patient Safety and Quality Improvement Act. This decision underscores the importance of adherence to regulatory standards for patient safety organizations.
The U.S. Department of Health and Human Services rescinds several documents under Executive Order 14192, aimed at deregulation. This move aligns with the administration's broader initiative to enhance government efficiency and governance.
HRSA has published a notice regarding petitions received under the National Vaccine Injury Compensation Program, fulfilling requirements of the Public Health Service Act. This program allows individuals to seek compensation for vaccine-related injuries, with the U.S. Court of Federal Claims overseeing the petitions.
HRSA extends the performance period for Pueblo of Jemez by five months, now ending on July 31, 2026. This extension ensures continued access to essential primary health care services for the community served by Pueblo of Jemez.
The Physician-Focused Payment Model Technical Advisory Committee (PTAC) will hold meetings in 2026 to discuss and vote on proposals for physician-focused payment models (PFPMs). These public meetings will address current and previously submitted PFPMs.
The NIH is seeking public comment on a proposed shift in embryonic stem cell research to prioritize patient-centric biotechnologies. This initiative aims to reduce reliance on human embryonic stem cells, reflecting a significant change in research focus.
Ilona Reischl has been re-elected as chair of the EMA's Committee for Advanced Therapies, which plays a crucial role in evaluating advanced therapy medicinal products (ATMPs). This leadership continuity is expected to support the evolving landscape of ATMP regulations.
Clinical trials for new Lyme disease vaccines are currently underway, but no vaccine is available yet. This highlights ongoing efforts to address Lyme disease prevention amid rising cases.
The CDC has released new guidelines addressing anemia and iron deficiency, emphasizing the need for improved screening and treatment protocols. These guidelines aim to enhance patient outcomes and reduce the prevalence of these conditions in vulnerable populations.
The CDC has updated its NoroSTAT data table, detailing suspected and confirmed norovirus outbreaks reported by state health departments. This resource aids in tracking norovirus activity across the U.S.
The MPN PROGRESSion Registry is launched to track symptoms, treatments, and disease progression in individuals with myeloproliferative neoplasms (MPNs). This observational study aims to enhance understanding of conditions like Polycythemia Vera and Essential Thrombocythemia.
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