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A total of 182 posts are filed under Research
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
Introducing the FirST-4-LEMS Study
Your participation can help change the lives of people with Lambert-Eaton Myasthenic Syndrome (LEMS.) As someone living with LEMS and… Continue Reading
Realities of Rare Disease and the Role of Patient Investors
For the EBD Group, Guest post by Nicole Boice, Founder & CEO, Global Genes – Allies in Rare Disease Rare Disease represents the largest… Continue Reading

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