Source: U.S. Food and Drug Administration
Updated December 3, 2018
Related
Amazon Web Services Commits $10 million to Pediatric Rare Disease Research & More — This Week in RARE Daily
This Week in RARE Daily is a feature from Global Genes where you can get a quick […]
Read moreThe need for regulators to exercise their flexibility to address ultra-rare therapies — 2024 NEXT Report
The path to rare disease drug development comes with built-in obstacles as it is, but when seeking […]
Read moreFDA Grants Sarepta Expanded Approval for DMD Gene Therapy & More — This Week in RARE Daily
This Week in RARE Daily is a feature from Global Genes where you can get a quick […]
Read moreImproved Gene Editing System Makes Gene-Sized Edits & More — This Week in RARE Daily
This Week in RARE Daily is a feature from Global Genes where you can get a quick […]
Read more2024 RARE Champions of Hope Nominees Announced
The RARE Champions of Hope Awards celebrate, honor and recognize those in the rare disease community who […]
Read morePenn Study of Rare Disease Sheds Light on More Common Ones & More — This Week in RARE Daily
This Week in RARE Daily is a feature from Global Genes where you can get a quick […]
Read moreFDA Approves Amgen’s Soliris Biosimilar & More — This Week in RARE Daily
This Week in RARE Daily is a feature from Global Genes where you can get a quick […]
Read moreCrafting business models to enable sustainable development of N-of-1 therapies — 2024 NEXT Report
When traditional pharma decides that therapies for N-of-1 and ultra-rare diseases is too risky, not-for-profits and public […]
Read moreFNIH Launches Partnership to Accelerate ALS Diagnosis and Treatment & More — This Week in RARE Daily
This Week in RARE Daily is a feature from Global Genes where you can get a quick […]
Read more