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Gene Therapy Net – News, Conferences, Vectors, Literature, Clinical Trials and more
- DMD Gene Therapy Candidate RGX-202 Earns Orphan Drug Status
- NIH and FDA Lead Coalition to Accelerate Gene Therapy Development for Rare Diseases
- FDA: Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations
- Fourth Boy Dies in Trial of Astellas Gene Therapy Candidate
- New gene-delivering workhorse could make gene therapy safer, more effective for muscle diseases
- Lysogene Announces First Patient in the United States Dosed with LYS-GM101 Investigational Gene Therapy for the Treatment of GM1 Gangliosidosis
- FDA allows Novartis gene therapy trials to resume after nearly 2 year pause
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