The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history— how disorders such as spinal muscle atrophy(SMA) run their course if untreated — the choice of “efficacy endpoints” in clinical trials, and how…
NORD
The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in Vienna said. More than 900 people from 58 nations attended the 9th European Conference on Rare Diseases & Orphan Drugs (ECRD),…
Recent Posts
- Using approved LOPD combination therapy at home is safe, study finds
- How a Pompe disease diagnosis both tested and transformed our marriage
- New Phase 2 trial tests oral add-on therapy for late-onset Pompe disease
- My journey of learning how to explain Pompe disease to others
- Early trial of late-onset Pompe disease drug yields positive results