Precision BioSciences Inc., a clinical-stage company developing gene editing therapies for diseases with high unmet need, has ...

The One Big Beautiful Bill Act takes a big step in the rare disease space by expanding the contours of the orphan drug ...

Orphan drug designation is granted by the FDA to products that prevent, diagnose or treat a rare disease or condition affecting fewer than 200,000 people in the U.S.

Orphan Drugs Strategic Research Report 2024-2030: A Beacon of Hope for Patients with Rare Diseases - Market to Reach $394.7 Billion by 2030 - ResearchAndMarkets.com ...

Cure Rare Disease Receives Orphan Drug Designation from FDA for Investigational Therapy Targeting Spinocerebellar Ataxia Type 3 (SCA3) Provided by Business Wire May 14, 2025, 4:00:00 AM ...

U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation and Rare Pediatric Disease designation for bexicaserin for the treatment of Dravet syndrome LA JOLLA, Calif.--(BUSINESS ...

Spurred by the Orphan Drug Act of 1983, there are 1303 US FDA-approved orphan drugs (ODs), with an estimated 7561 in development and 6.9 billion US dollars in federal funding for rare disease ...

Orphan Drug and Rare Pediatric Disease designations follow U.S. FDA acceptance of IND application for ABO-101 for the treatment of PH1, with Phase 1/2 trial initiation anticipated in H1 2025.

Rare diseases suffer from a myriad of challenges, including misdiagnosis, no diagnosis, or lack of appropriate treatments. The NIH estimates that ~10,000 rare diseases are affecting ~ 30 million ...

Amy is one of many patients meant to benefit from the Orphan Drug Act of 1983, which aims to encourage pharmaceutical companies to do more to treat rare diseases—like different types of ...

Dr Makary and colleagues note in their article that the 1983 ODA was designed to incentivize drug makers to develop drugs for rare or "orphan" diseases that affect fewer than 200,000 people in the ...