• The Give Kids a Chance Act – Reinstates the rare pediatric priority review voucher program, also known as the Creating Hope ...
The FDA has approved Evrysdi in tablet form for patients with spinal muscular atrophy aged 2 years and older who weigh more ...
NEW YORK - OS Therapies, Inc. (NYSE-A: OSTX), a small-cap biotech firm with a market capitalization of $42 million focusing on cancer treatment solutions, has announced the completion of its Phase 2b ...
Research and development expenses were $12.6 million for the second quarter of fiscal 2025, compared with $12.0 million for the same period a year ago, with the increase primarily attributable to ...
The 5 mg risdiplam tablet provides the same efficacy and safety for spinal muscular atrophy as the currently available oral ...
Adult and pediatric patients with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas that are not amenable ...
GOMEKLI is the first and only medicine approved for both adults and children with NF1-PN –– Approval based on positive data ...
Stamford-based SpringWorks Therapeutics, a commercial-stage biotech company developing treatments for rare diseases and ...
Moleculin Biotech (MBRX) has received first country regulatory approval in Europe to begin recruiting for its Phase 3 pivotal trial protocol ...
The FDA has made some modest efforts, but it needs an internal organization that understands and would be an aggressive ...
BD affects millions but remains misunderstood, underdiagnosed, and underfunded. Researchers are working to change this by ...
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