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A total of 288 posts are filed under Business
Express Scripts Program Designed to Meet Needs of Payers, Not Patients
At the end of May, the pharmacy benefits manager Express Scripts announced a new program it said was aimed at “reducing the burden on… Continue Reading
New Bluebird Bio Data Shows Gene Therapy Promising for Thalessemia
Rare Daily Staff Bluebird Bio said data from its phase 1/2 Northstar study of its experimental LentiGlobin gene therapy in adolescents and… Continue Reading
Experimental Drug for “Elephant Man” Disease May Benefit Certain Cancers Too
Joseph Merrick, who was known as the Elephant Man, suffered from Proteus syndrome, a rare disorder that causes abnormal and disproportionate… Continue Reading
BridgeBio Pharma Launches CoA Therapeutics to Target Coenzyme-A for Rare Developmental Disorders
Rare Daily Staff BridgeBio Pharma said it launched CoA Therapeutics, a biopharmaceutical company that will develop small-molecules designed… Continue Reading
Inozyme Pharma Offers No-Cost Genetic Testing for Rare Calcification Disorders
Rare Daily Staff Inozyme Pharma has introduced a no-cost, third-party genetic testing program designed to improve detection and… Continue Reading
FDA Grants Rare Pediatric Disease Designation to Albireo’s Candidate for Rare Liver Disease
Rare Daily Staff The U.S. Food and Drug Administration granted Albireo Pharma’s lead candidate A4250 Rare Pediatric Disease designation… Continue Reading
Alexion and Complement Pharma Form Partnership to Co-Develop Complement Inhibitor for Neurodegenerative Disorders
Rare Daily Staff Alexion Pharmaceuticals entered into a partnership with Complement Pharma to co-develop the preclinical C6 complement… Continue Reading

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