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A total of 421 posts are filed under Business
Abeona Therapeutics Appoints New CMO
Rare Daily Staff Abeona Therapeutics said it appointed João Siffert, an industry veteran with medical and academic experience, as head of… Continue Reading
AveXis Submits SMA Gene Therapy for Regulatory Approval in U.S., Europe, and Japan
Rare Daily Staff AveXis, in a letter addressed to the spinal muscular atrophy community, said it submitted regulatory applications in the… Continue Reading
SMA Drug Developers Share $3 Million Breakthrough Prize
Rare Daily Staff Ionis Pharmaceuticals pharmacologist Frank Bennett and Cold Spring Harbor Laboratory Biochemist Adrian Krainer will share a… Continue Reading
Pharnext Reports Positive Topline Results from Late-Stage Charcot-Marie-Tooth Trial
Rare Daily Staff Pharnext reported positive topline results from its pivotal late-stage clinical trial evaluating two doses of its… Continue Reading
EMA Validates Rigel’s Application for ITP Drug
Rare Daily Staff The European Medicines Agency validated the Marketing Authorization Application for Rigel Pharmaceuticals’ fostamatinib… Continue Reading
Ionis Enters Collaboration with Roche Worth up to $760 Million
Rare Daily Staff Ionis Pharmaceuticals said it entered into a collaboration with Roche to develop IONIS-FB-LRx for the treatment of… Continue Reading
Therachon Expands Rare Disease Pipeline with Acquisition of GLyPharma
Rare Daily Staff Rare disease drug developer Therachon said it would acquire GLyPharma Therapeutic for an undisclosed amount, expanding its… Continue Reading

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