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A total of 184 posts are filed under Research
Medgadget: Future of Global Rare Disease Treatment Market by 2016 – 2024
It is estimated that, approximately 5% of the global population suffer from a rare disease and half of the global population affected by… Continue Reading
Abeona Therapeutics Receives Orphan Drug Designation in The European Union for ABO-201 Gene Therapy Program in Juvenile Batten Disease
  Abeona Therapeutics Inc. (NASDAQ: ABEO), a clinical-stage biopharmaceutical company focused on developing therapies for… Continue Reading
World’s Largest-Ever Study of SYNGAP1 (MRD5) Launches
Bridge the Gap – SYNGAP Education and Research Foundation has launched the largest-ever study to research SYNGAP1 that causes… Continue Reading
SPINRAZA™ (nusinersen) Approved in U.S. to Treat Broad Range of Patients with Spinal Muscular Atrophy
– First Treatment Approved for SMA –– SPINRAZA Improved Motor Function in Patients with SMA; Greater Percentage of Infantile-Onset… Continue Reading
First Patient in Pivotal Trial of BHV-4157 in Patients With Hereditary Spinocerebellar Ataxia
Biohaven Pharmaceutical Holding Company Ltd. (“Biohaven” or the “Company”) announced today that it has enrolled the first patient… Continue Reading
RDR: FDA Approves Rubraca (rucaparib) for Rare Ovarian Cancer
The FDA has approved the orphan drug Rubraca (rucaparib) to treat women with advanced ovarian cancer who have been treated with 2 or more… Continue Reading
CureDuchenne Ventures Invests in TRiNDS to Provide Clinical Trial Solutions for Neuromuscular Disorders
CureDuchenne Ventures, LLC, a company that funds research to find a cure for Duchenne muscular dystrophy, announced today that they… Continue Reading

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