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A total of 519 posts are filed under Innovation
Enzyvant Begins Rolling Submission for FDA Approval of Tissue-based Therapy for Rare Immune Deficiency
Rare Daily Staff Enzyvant said that it has started a rolling submission with the U.S. Food and Drug Administration for approval to market… Continue Reading
Sanofi Genzyme Creates Challenge to Find Digital Technologies to Speed Rare Disease Diagnoses
Rare Daily Staff Sanofi Genzyme has issued a call for submissions for its Rare Innovation Challenge, an effort to find innovative digital… Continue Reading
U.K. Gene Therapy Manufacturing Consortium Seeks to Address Barrier to Commercialization
Rare Daily Staff Plasticell, a developer of stem cell technologies and therapies, is leading a consortium of United Kingdom gene therapy… Continue Reading
Summit’s DMD Therapy Fails in Trial; Company Discontinues Development
Rare Daily Staff Summit Therapeutics said it would discontinue development of its experimental therapy to treat the progressive, rare, and… Continue Reading
First Marijuana-Derived Prescription Drug Wins FDA Approval for Rare Epilepsies
Rare Daily Staff The U.S. Food and Drug Administration approved GW Pharmaceuticals Epidiolex for the treatment of seizures associated with… Continue Reading
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
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

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