Rare Leader: Dawn Ireland, President, CDH International

The Basics Name: Dawn Ireland Title: President…

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
Sanofi Genzyme and PerkinElmer Expand Access to Free Diagnostic Testing
Rare Daily Staff The Lantern Project, a program Sanofi Genzyme created with PerkinElmer Genomics, is offering no-cost testing for people… Continue Reading
Making the Case for an FDA Rare Disease Center of Excellence
Two attorneys specializing in regulatory law recently proposed the creation of an FDA Rare Disease Center of Excellence to better allow the… 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
Rare Leader: Marc Yale, Executive Director, IPPF
The Basics Name:  Marc Yale Title:  Executive Director Organization: International Pemphigus & Pemphigoid Foundation Disease focus:… Continue Reading
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