Daniel S. Levine

Daniel S. Levine is editor of Rare Daily. You can contact him by sending email to editor@globalgenes.org

Sarepta Reports Encouraging Preliminary Results in DMD Gene Therapy Study
Rare Daily Staff Sarepta Therapeutics said preliminary results from its phase 1/2a clinical trial of its experimental micro-dystrophin gene… Continue Reading
PTC Reports Positive Updated Interim Data for Experimental Drug to Treat SMA Type 1
Rare Daily Staff PTC Therapeutics said updated interim data from the first part of its FIREFISH study investigating its experimental drug… Continue Reading
FDA Removes Clinical Hold on Solid Biosciences Experimental DMD Gene Therapy
Rare Daily Staff The U.S. Food and Drug Administration has lifted the clinical hold on Solid Biosciences’ phase 1/2 clinical trial for its… Continue Reading
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
Rare Leader: James O’Brien, President of Prader-Willi Syndrome Australia
The Basics Name: James O’Brien Title/Organization: President, Prader-Willi Syndrome Australia; Founding Director, PWS Better Living… Continue Reading
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