Related
Ascendis Reports Positive Results for Experimental Achondroplasia Therapy
Rare Daily Staff Ascendis Pharma reported positive topline data from its pivotal double-blind placebo-controlled trial of its […]
Read moreEnabling On-Demand Therapies
Earlier this year, the company gained attention when it reported that developed a customized antisense oligonucleotide to […]
Read moreChiesi Global Rare Diseases Launches Grant Initiative to Support Research into LSDs
Rare Daily Staff Chiesi Global Rare Diseases has launched Find For Rare, a new grant initiative to provide […]
Read moreMDA Launches Program to Develop Gene Therapies for Ultra-Rare Neuromuscular Diseases
Rare Daily Staff The Muscular Dystrophy Association said it has launched the MDA Kickstart program for Ultra-Rare […]
Read moreFDA Grants Full Approval to Travere for Filspari in IgA Nephropathy
Rare Daily Staff The U.S. Food and Drug Administration granted Travere Therapeutics full approval and expanded use […]
Read moreAfter Driving an N-of-1 Therapy for Her Son, an Advocate Turns to Helping Others
Yiwei She, founder of the TNPO2 Foundation, discusses how her family was able to treat her son […]
Read moreStudy Explores Quality of Life Effects of Healthcare Access on Caregivers
Rare Daily Staff New strategies are needed to better support rare disease caregivers and improve health outcomes […]
Read moreNIMH Awards PsychoGenics $3 million Grant to Use AI Platform for Repurposing
Rare Daily Staff The National Institutes of Health’s National Institute of Mental Health awarded a Fast Track […]
Read morePutting Rare Disease Organization in the Business of Drug Development
Rare disease patient organizations are increasingly driving the discovery and development of therapies to treat the conditions […]
Read more