NEW DELHI — Recuperating in the cardiac care unit of Batra Hospital, 20-month-old Stawan Bijal Shah refuses his feed and cries for his mother. He is one of the youngest patients to undergo a coronary artery bypass surgery for a rare disease which damaged his heart. When Stawan was just...
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For the first time since 2020, EURORDIS (Rare Diseases Europe) recently held its Black Pearl Awards ceremony in person, recognizing the exceptional work and accomplishments of those who make a difference in the rare disease community.
Edinburgh, Scotland – A groundbreaking 25-year research program has unveiled key insights into how our brains age and what factors influence cognitive performance throughout life. The findings, published on 7 November 2024 in Genomic Psychiatry, draw from the Lothian Birth Cohorts (LBC) studies, which uniquely tracked participants’ cognitive abilities from...
ABOUT THE VIRTUAL GALA November 6, @ 8 PM EDT Presented by the Kissam Family and Mark & Rachel Rohr Family Foundation. Be inspired by stories from our community, hear from IRSF leadership, learn about the incredible work that our foundation is doing to accelerate research and empower families. We’re especially...
CAMBRIDGE, Mass. – 2seventy bio, Inc., (NASDAQ: TSVT), an emerging immuno-oncology company today announced its official launch as an independent, publicly traded company. 2seventy bio will trade on the Nasdaq Global Select Market, commencing tomorrow, November 5 under the ticker symbol “TSVT.” “We’ve done the intense work to reach the start...
Bloomington, MN – Pandemic-era changes may have permanently altered the clinical trial landscape as decentralized clinical trials gain broader acceptance. During COVID-19, more than three-quarters (76%) of pharmaceutical companies, device manufacturers and contract research organizations conducted off-site clinical trials. These types of trials specifically provide increased opportunity for individuals with...
EMERYVILLE, Calif. — 4D Molecular Therapeutics (Nasdaq: FDMT), a leading clinical-stage genetic medicines company focused on unlocking the full potential of genetic medicines to treat large market diseases, today announced an update on its regulatory interactions and development path for 4D-710, an aerosolized genetic medicine for the treatment of CF...
EMERYVILLE, Calif. — 4D Molecular Therapeutics, a leading clinical-stage genetic medicines company focused on unlocking the full potential of genetic medicines to treat large market diseases in ophthalmology and pulmonology, today announced that the U.S. Food and Drug Administration (FDA) has granted the RMAT designation for the investigational genetic medicine...
EMERYVILLE, Calif. — 4D Molecular Therapeutics, a leading clinical-stage genetic medicines company focused on unlocking the full potential of genetic medicines to treat large market diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) for the Company’s product candidate aerosolized 4D-710...
Washington DC – As of Dec. 22, the FDA had approved at least 88 novel treatments in 2023, for indications ranging from breast and colorectal cancer to Duchenne muscular dystrophy to sickle cell disease. Rare diseases such as Rett syndrome and Friedreich’s ataxia saw their first approvals. Some decisions, like Sarepta’s...