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Drug Development

A total of 263 posts are filed under Drug Development
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
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
The What and the How
I tend to multi-task, thanks to a little help with some modest technology. During any given day I will have a long to-do list on my computer… Continue Reading
FDA Approves Leadiant Biosciences’ Enzyme Replacement Therapy for ADA-SCID
Rare Daily Staff The U.S. Food and Drug Administration approved Revcovi, Leadiant Biosciences’ enzyme replacement therapy for the… Continue Reading
EMA Grants PRIME Designation to Orchard Therapeutics’ Beta-Thalassemia Gene Therapy
Rare Daily Staff The European Medicines Agency granted Orchard Therapeutics Priority Medicines (PRIME) designation to OTL-300, an… Continue Reading
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