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Science & Technology

A total of 129 posts are filed under Science & Technology
Penn Medicine Explores ResearchKit’s Potential for Studying Rare Disease
[SOURCE: Mobi Health News] In a first for the University of Pennsylvania Health System, Penn Medicine launched a new ResearchKit app today… Continue Reading
Lysogene Receives Rare Pediatric Disease Designation from FDA for LYS-GM101 for Treatment of GM1 Gangliosidosis
Lysogene, a leading, clinical-stage biotechnology company specializing in gene therapy for rare central nervous system diseases, today… Continue Reading
RDR: Generic Xyrem to Treat Narcolepsy Gets FDA Approval
The FDA has approved a generic version of Xyrem (sodium oxybate) Oral Solution, to treat cataplexy and excessive daytime sleepiness in… 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
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
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