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Drug Development

A total of 44 posts are filed under Drug Development
Sobi Initiates Late-Stage Study of Kineret to Treat Still’s Disease
Rare Daily Staff Swedish Orphan Biovitrum said that it has randomized the first patient in its late-stage study of Kineret in the treatment… Continue Reading
Ultragenyx Wins FDA Approval of First Therapy for MPS VII
Rare Daily Staff The U.S. Food and Drug Administration approved Ultragenyx Pharmaceutical’s MEPSEVII, the first treatment for children and… Continue Reading
Naia Rare Diseases Gets Green Light for Clinical Trials of Treatment for Short Bowel Syndrome
Rare Daily Staff Naia Rare Diseases, a biopharmaceutical company developing drugs for rare gastrointestinal diseases, said it has received… Continue Reading
Akcea Initiates Mid-Stage Trial of Therapy in Patients with Rare Hyperlipidemias
Rare Daily Staff Akcea Therapeutics, an affiliate of Ionis Pharmaceuticals focused on developing and commercializing drugs to treat patients… Continue Reading
Synlogic Reports Positive Results in Early-Stage Study of Treatment for UCD
Rare Daily Staff Synlogic reported positive top-line clinical data from its early-stage trial of experimental therapeutic SYNB1020 as a… Continue Reading
Horama raises $22 million to Advance Gene Therapy Programs for Eye Disease
Rare Daily Staff French biotech Horama, which is developing gene therapies for the treatment of rare, inherited retinal diseases, said it… Continue Reading
Alnylam Initiates Late-Stage Study of RNAi Therapeutic for AHPs
Rare Daily Staff Alnylam Pharmaceuticals said it initiated a late-stage study of givosiran, a subcutaneously administered, experimental RNAi… Continue Reading
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