Rare disease news, mapped to the diseases behind the headlines. AI-classified across pipeline, policy, funding, science, and community.
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The FDA has determined the regulatory review period for EXBLIFEP, which is necessary for patent extension applications. This notice is part of the legal requirements for patent claims related to human drug products.
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 HHS is undergoing a review under the Paperwork Reduction Act, which may impact data collection processes. This review is part of ongoing efforts to streamline agency operations.
The CDC is seeking public comment on a proposed information collection project related to the National Network of Sexually Transmitted Diseases Clinical Prevention Training Centers. This initiative aims to enhance data collection while minimizing public burden, as mandated by the Paperwork Reduction Act.
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.
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 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'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 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 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 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 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.
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.
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.
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