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 781–800 of 2615 stories
The EMA's Pharmacovigilance Risk Assessment Committee (PRAC) held its monthly meeting to discuss ongoing risk management responsibilities related to medicine safety. Key statistics and agenda items were reviewed, but no significant decisions or updates were reported.
AFM-Téléthon 2025 is set to raise awareness and funds for rare diseases, continuing its legacy of supporting research and patient advocacy. The event aims to engage the community and highlight the importance of rare disease initiatives.
EURORDIS announces the ERN ReCONNET event focused on sharing good clinical practices for rare diseases in 2025. This initiative aims to enhance collaboration among stakeholders in the rare disease community.
EURORDIS hosts a webinar focusing on the UnDiagnosed Patients Working Group under ERN LUNG, aiming to enhance collaboration and support for patients with undiagnosed lung diseases. This initiative highlights the importance of patient engagement in rare disease research and advocacy.
The European Commission has extended the deadline for applications to participate in EMA’s Paediatric Committee, now due by December 5, 2025. This initiative aims to include civil society representatives from patient and healthcare professional organizations.
EURORDIS highlights the HLM RARE initiative aimed at improving the lives of individuals with rare diseases. The initiative focuses on raising awareness and advocating for better policies and support systems.
The European Conference on Rare Diseases and Orphan Products 2026 is set to take place, focusing on advancements and advocacy in the rare disease community. This event aims to bring together stakeholders to discuss critical issues affecting patients and the development of orphan products.
FDA approves Novartis' Itvisma, an intrathecal formulation of onasemnogene abeparvovec, for spinal muscular atrophy in patients aged 2 and older. The STEER trial showed a significant 2.39-point improvement in motor function compared to sham treatment, marking a pivotal advancement in gene therapy for neurological disorders.
The U.S. Food and Drug Administration today approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of spinal muscular atrophy (SMA) in adult and pediatric patients 2 years of age and older with confirmed mutation in the survival motor neuron 1 (SMN1) gene.
EMA recommends granting marketing authorization for Teizeild (teplizumab) to delay the onset of stage 3 type 1 diabetes in adults. This first-in-class treatment represents a significant advancement in diabetes management.
The U.S. Food and Drug Administration today announced a pilot program designed to streamline communications with sponsors following formal meetings.
EMA's Emergency Task Force is enhancing its scientific advice process for medicines targeting public health threats, including antimicrobial resistance. This new approach aims to provide clearer guidance on clinical trial design, facilitating faster development and approval of critical therapies during health emergencies.
The U.S. Food and Drug Administration today announced it is taking action to approve new labeling submitted by the company that includes the addition of a Boxed Warning, the agency’s most prominent safety warning, to Elevidys (delandistrogene moxeparvovec-rokl), and that the indication section of th
NORD has launched a new Request for Proposals (RFP) to implement two patient registries on the IAMRARE® platform, funded by RDCA-DAP®. This initiative aims to enhance data collection and support for rare disease communities.
Over 110 individuals affected by rare diseases gathered in Milwaukee for the 'Living Rare, Living Stronger' event, fostering connections among patients, caregivers, and advocates. This initiative highlights the importance of community support in navigating the challenges of rare diseases.
The EMA's CHMP recommended ten new medicines for approval during its November 2025 meeting, indicating a positive trend in drug development. Specific details on the drugs and their indications were not disclosed in the highlights.
The U.S. government has reopened following the signing of a continuing resolution by the President on November 12. This agreement funds most government operations, ensuring stability for ongoing health policies and programs.
The U.S. Department of Health and Human Services (HHS) and the U.S. Food and Drug Administration (FDA) today announced that Richard Pazdur, M.D., has been appointed director of the Center for Drug Evaluation and Research (CDER). Pazdur is a 26-year veteran of the FDA.
The U.S. Department of Health and Human Services (HHS) today announced historic action to restore gold-standard science to women’s health.
The EU's upcoming 2028-34 budget will significantly impact funding for rare diseases, with decisions in the coming months crucial for resource allocation. Stakeholders are urged to advocate for improved financial frameworks to support those living with rare conditions.
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.