Related
Broken String Enters Collaboration with Francis Crick Institute to Advance ALS Research
Rare Daily Staff Genomics company Broken String Biosciences said that it has entered a research collaboration with […]
Read morePfizer Pauses Enrollment in DMD Gene Therapy Study After Patient Death
Rare Daily Staff Pfizer, in a May 7 letter to Duchenne muscular dystrophy patient advocacy organizations, reported that […]
Read moreStealth Biotherapeutics’ Barth Syndrome NDA Receives FDA Priority Review
Rare Daily Staff The U.S. Food and Drug Administration has granted Priority Review to Stealth Biotherapeutics’ New […]
Read moreEledon Raises $50 Million in Private Placement
Rare Daily Staff Eledon Pharmaceuticals entered into a securities purchase agreement with certain institutional and accredited healthcare […]
Read moreZenas Raises $200 Million to Advance Immunology-Focused Clinical Programs
Rare Daily Staff Zenas BioPharma, a company developing and commercializing inflammation and immunology-directed therapies for rare and […]
Read moreFDA Grants Fast Track Designation to Iecure for Treatment of Neonatal OTC Deficiency
Rare Daily Staff The U.S. Food and Drug Administration granted Fast Track Designation to Iecure ECUR-506, the […]
Read moreRare Disease Focused Therapeutics M&A Soars in April
Two sizable acquisitions marked an otherwise slow April for rare disease focused therapeutics financings and deals, with […]
Read moreGlycoMimetics’ Uproleselan Fails Pivotal Phase 3 Study in Relapsed/Refractory AML
Rare Daily Staff GlycoMimetics said the phase 3 global pivotal study of its experimental treatment for relapsed/refractory […]
Read moreResearchers Develop Global Approach for Using AI to Identify Kids with Rare Diseases
Rare Daily Staff Diagnosing children with rare, genetic diseases often involves misdiagnoses and prolonged diagnostic odysseys but […]
Read more