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A total of 163 posts are filed under Research
FDA Approves Lundbeck’s IV Carbamazepine For Certain Seizures
By James Radke, PhD at Rare Disease Report The FDA has approved Carnexiv (carbamazepine) injection as a short-term replacement therapy for… Continue Reading
Abeona Therapeutics Authorized to Move Forward with Clinical Trial for Sanfilippo Syndrome Treatment
Abeona Therapeutics Inc. (NASDAQ: ABEO), a clinical-stage biopharmaceutical company focused on developing gene therapies for… Continue Reading
New App for Cystic Fibrosis Patients Helps to Manage Symptoms
People with cystic fibrosis (CF) need help to ensure they are getting correct nutrition and the right amount of enzymes. They also need… Continue Reading
FDA Approves Vertex’s Orkambi for Cystic Fibrosis Children 6-11 Years Old
by Andrew Black for Rare Disease Report The FDA has approved Vertex Pharmaceuticals’ drug Orkambi (lumacaftor/ivacaftor) for use in… Continue Reading
New Grant Available for Projects Dealing in Eosinophil-Associated Diseases
Objective: The APFED HOPE Pilot Grant was established to foster the development of new projects relevant to eosinophil-associated diseases… Continue Reading
Color Genomics Raises $45M in Series B. With a Little Help From Bono
Yep, you read that correctly. Maybe the U2 front man has finally found what he’s looking for (sorry) by investing in cancer testing… Continue Reading
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New Study Investigates “Touch Blindness” as Potential Genetic Mutation
Two young patients with a rare condition have helped scientists unlock a better understanding of our sense of touch and what researchers… Continue Reading