Rare disease news, mapped to the diseases behind the headlines. AI-classified across pipeline, policy, funding, science, and community.
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NORD calls on Congress to swiftly reauthorize the Rare Pediatric Disease Voucher Program, which has facilitated 63 FDA vouchers for 47 rare pediatric diseases, including 43 with no prior treatments. This program is crucial for advancing therapies for underserved children.
Today, the U.S. Food and Drug Administration conditionally approved Exzolt Cattle-CA1 (fluralaner) topical solution for the prevention and treatment of New World screwworm (NWS) larval infestations, and the treatment and control of cattle fever tick in beef cattle 2 months of age and older and repl
The Journée Annuelle de l’AIRG in France highlights ongoing efforts in rare disease advocacy. This annual event fosters collaboration among stakeholders to improve awareness and support for rare disease patients.
The U.S. Food and Drug Administration (FDA) today announced that Tracy Beth Høeg, M.D., Ph.D., has been appointed acting director of the Center for Drug Evaluation and Research (CDER).
The U.S. Food and Drug Administration today granted approval for Avance (acellular nerve allograft-arwx) in surgical implantation.
The National Organization for Rare Disorders (NORD) has expanded its network by adding seven new Rare Disease Centers of Excellence, bringing the total to 46 institutions. This initiative aims to enhance rare disease care and foster research collaboration across the country.
The U.S. Food and Drug Administration, in coordination with the U.S. Department of Justice, announced today that the U.S. Marshals Service seized approximately 73,000 units of 7-hydroxymitragynine (7-OH) products—valued at roughly $1 million—from three firms in Missouri.
Today, the U.S. Food and Drug Administration (FDA) issued draft guidance outlining specific product types for which the FDA believes six-month non-human primate toxicity testing can be eliminated or reduced.
The House of Representatives has passed the Give Kids a Chance Act (H.R. 1262), which aims to enhance policies that support rare disease research and treatment. The EveryLife Foundation for Rare Diseases commends this legislative progress as a significant step towards improving outcomes for affected children.
The U.S. Food and Drug Administration today announced the deployment of agentic AI capabilities for all agency employees.
The TSC Alliance and Dup15q Alliance emphasize the critical role of awareness in improving early diagnosis and outcomes for infantile spasms during Awareness Week. Their collaboration aims to educate families on symptoms and the importance of timely intervention.
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.
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