Rare disease news, mapped to the diseases behind the headlines. AI-classified across pipeline, policy, funding, science, and community.
Be among the first to receive Kisho Intelligence Briefings
Showing 461–480 of 2553 stories
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.
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.
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 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.
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's NoroSTAT provides weekly updates on suspected and confirmed norovirus outbreaks across states, enhancing public health monitoring. This data is crucial for informing policy decisions and outbreak response strategies.
The CDC has released new guidelines aimed at improving osteoporosis prevention and management. These guidelines emphasize the importance of early screening and treatment to reduce fracture risk among at-risk populations.
The CDC has released quarterly provisional estimates for selected birth indicators for Q3 2023. These estimates provide insights into natality trends, which may inform health policy and resource allocation.
The CDC has released technical notes regarding quarterly provisional estimates for natality in Q3 2025. This update provides insights into birth rates but lacks significant policy implications.
The FDA has released draft guidance for cuffless non-invasive blood pressure measuring devices, outlining recommendations for clinical performance testing. This guidance is currently not final and is intended to support premarket submissions.
The FDA has issued a 5-year debarment order against Tyler Jordan Hall for importing unapproved drugs into the U.S. This action underscores the agency's commitment to enforcing drug approval regulations.
The Federal Register has published updates regarding the Sickle Cell Advisory Committee, including amendments and renewals. This committee plays a crucial role in advising on policies related to sickle cell disease.
The Administration for Children and Families seeks a 3-year extension for the Congressionally Directed Community Projects information collection, with revisions aimed at simplifying application requirements. This extension is set to expire on March 31, 2026.
The Advisory Council on Alzheimer's Research, Care, and Services will hold its first meeting of 2026 to introduce new members and discuss strategies to alleviate the burden of Alzheimer's disease. This meeting aims to enhance collaboration among federal agencies in addressing Alzheimer's and related dementias.
The Administration for Children and Families proposes to resume data collection for Tribal Lead Agencies seeking to use Child Care and Development Funds for facility construction or renovation. This initiative aims to streamline the process and reduce administrative burden.
As of January 2025, the FDA has approved only one orphan drug class for alpha-1 antitrypsin deficiency (AATD) since 1987, despite 20 designations. The EMA has granted nine orphan designations for AATD but has yet to approve any.
A CDC report highlights the prevalence of violence perpetration among men aged 18-24 in low- and middle-income countries, linking it to childhood exposure to violence. This data may inform future policy decisions aimed at violence prevention and youth support programs.
The CDC reports on the performance of the CureTB program from 2016 to 2023, focusing on continuity of care for tuberculosis patients relocating internationally. This evaluation highlights the program's impact on patient management and health outcomes.
The NIH has announced a significant policy shift to end the use of human fetal tissue in NIH-supported research, redirecting focus towards next-generation validated research models. This change reflects evolving ethical standards and may impact future research funding and methodologies.
The United States has officially withdrawn from the World Health Organization, citing failures in handling COVID-19 and a desire to focus on bilateral health initiatives. This decision may impact global health collaboration and funding.
Get the week's most important rare disease developments — pipeline moves, policy changes, and funding signals — delivered to your inbox every Monday.
Free weekly briefing. Unsubscribe anytime.