Related
FDA Lifts Partial Clinical Hold on IND for EryDel’s Phase 3 Therapy for Ataxia-Telangiectasia
Rare Daily Staff Quince Therapeutics said that The U.S. Food and Drug Administration has lifted the partial […]
Read moreFDA Approves Amicus’s New Treatment for Adults with Late-Onset Pompe Disease
Rare Daily Staff The U.S. Food and Drug Administration has approved Amicus Therapeutics’ Pombiliti + Opfolda, a […]
Read moreIonis’s Eplontersen Phase 3 Results Show Consistent and Sustained Benefit in Patients with ATTRv-PN
Rare Daily Staff Ionis Pharmaceuticals reported that The Journal of the American Medical Association published positive results […]
Read morePTC Cuts 25 Percent of Workforce in Further Strategic Prioritization
Rare Daily Staff Rare disease focused biotech PTC Therapeutics announced further strategic prioritization and an associated workforce […]
Read moreCapricor Raises $23 Million in Registered Direct Offering
Rare Daily Staff Capricor Therapeutics said it has raised $23 million, which it will use to fund […]
Read moreFDA Expands Approved Uses of Pfizer Drug to Include Childhood Leukemia
Rare Daily Staff The U.S. Food and Drug Administration expanded the approval of Pfizer’s Bosulif to include […]
Read moreThéa Terminates Plans to Acquire ProQR Assets
Rare Daily Staff RNA therapy developer ProQR Therapeutics said that Laboratoires Théa’s previously announced agreement to acquire […]
Read moreSoleno Therapeutics Raises $120 Million After Reporting Positive Prader-Willi Trial Results
Rare Daily Staff Two days after reporting positive top-line results of its experimental treatment for Prader-Willi syndrome, […]
Read moreFDA Grants Priority Review for Merck’s Sotatercept to Treat Adults with Pulmonary Arterial Hypertension
Rare Daily Staff The U.S. Food and Drug Administration (accepted for priority review a new Biologics License […]
Read more