Rocket boosted by FDA alignment on pivotal gene therapy trial
Por um escritor misterioso
Last updated 10 março 2025

Rocket Pharmaceuticals has reach | Rocket Pharmaceuticals has reached alignment with the FDA on the design of a pivotal phase 2 rare disease trial, positioning it to run a 12-patient study that could support accelerated approval of a gene therapy.

acute myeloid leukemia

Rocket boosted by FDA alignment on pivotal gene therapy trial
Thinkeen Global

Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial - The Lancet Neurology

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CCS_Book of abstracts - 2023 - Haemophilia - Wiley Online Library

PDF) Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord

ovid-ex991_7.pptx.htm
Susan B. Nichols on LinkedIn: Rocket boosted as FDA alignment on pivotal gene therapy trial design sends…

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