Rare disease news, mapped to the diseases behind the headlines. AI-classified across pipeline, policy, funding, science, and community.
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The CDC highlights the impact of cerebral palsy (CP) on movement, balance, and posture, emphasizing the need for increased awareness and advocacy. This group of disorders affects many individuals, necessitating ongoing support and resources.
The CDC outlines its commitment to public health and fiscal responsibility, emphasizing transparency and accountability to taxpayers. This initiative aims to enhance public trust and ensure effective use of resources.
The CDC provides an overview of cerebral palsy, detailing symptoms, causes, and treatment options. This resource aims to enhance awareness and understanding of the condition among patients and healthcare providers.
The CDC highlights key risk factors for congenital cerebral palsy and emphasizes prevention strategies before, during, and after birth. This information is crucial for healthcare policies aimed at reducing incidence rates.
The CDC has released new guidelines focusing on older adult health, emphasizing preventive care and chronic disease management. These recommendations aim to improve health outcomes for seniors across the United States.
The Indian Health Service has approved reimbursement rates for inpatient and outpatient medical care for Calendar Year 2026. This decision impacts funding and access to healthcare services within IHS facilities.
The FDA has released draft guidance on its authority to access records related to cosmetic products, aimed at clarifying the criteria and process for industry compliance. This document addresses frequently asked questions and outlines the agency's current expectations.
The FDA is seeking public comment on a citizen petition from Celiac Journey, which calls for stricter labeling requirements for gluten in packaged foods. The petition requests that all gluten-containing ingredients be explicitly listed and that cross-contact controls be implemented to protect consumers with celiac disease.
The NIH Office of the Director is seeking public comment on a proposed data collection project related to the Genetic Testing Registry. This initiative aligns with the Paperwork Reduction Act, allowing stakeholders to provide input before submission to the OMB.
A recent study published in PLOS Genetics highlights the potential of large, diverse population cohorts, such as the All of Us Data, to uncover insights into rare diseases. This research emphasizes the importance of inclusivity in genomic studies to enhance understanding and treatment of rare conditions.
Genespire plans to advance its single-dose gene therapy GENE202 for methylmalonic acidaemia to clinical trials by the end of 2026. This follows a growing interest in therapies for this rare metabolic disease, with Moderna's mRNA-3705 currently being the only drug in development.
The FDA reports positive outcomes from its ImportShield Program, initiated in August 2025, aimed at enhancing oversight of imported products at U.S. ports. This program is part of ongoing efforts to modernize FDA review processes.
The FDA has issued a Request for Information to enhance gluten ingredient disclosure and prevent cross-contact in packaged foods. This initiative aims to improve safety for individuals with gluten-related disorders.
Congress has included a five-year reauthorization of the Rare Pediatric Disease Priority Review Voucher (PRV) program in a draft appropriations package, responding to advocacy from the rare disease community. This reauthorization aims to incentivize the development of treatments for pediatric rare diseases.
The ECRD 2026 event will focus on advancing rare disease policies across six thematic tracks, addressing the evolving landscape in Europe. This initiative aims to enhance collaboration and advocacy for rare diseases.
The Childhood Dementia Initiative emphasizes the need for unified terminology to enhance understanding and advocacy for childhood dementia and other rare diseases. This initiative aims to foster collaboration within the global rare disease community.
Carmen Purdel has been elected as the new chair of the EMA's Committee for Herbal Medicinal Products (HMPC). The HMPC is crucial for harmonizing the regulation of herbal medicinal products across the EU, ensuring high-quality scientific recommendations.
The CDC has released new guidelines for the use of Electronic Health Records (EHRs) to improve data sharing and patient care. These guidelines aim to enhance interoperability and ensure better access to health information across systems.
The CDC has released the latest National Diabetes Statistics Report, providing updated statistics on diabetes prevalence and impact. This periodic publication is crucial for informing health policy and resource allocation.
The CDC's National Healthcare Safety Network continues to be the leading system for tracking healthcare-associated infections across the U.S. This initiative underscores the importance of infection control in healthcare settings.
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