October 20, 2021
Stealth Bio Receives Refusal to File Letter from the FDA for Barth Syndrome Therapeutic
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October 20, 2021
FDA Grants Fast Track Designation to Avidity AOC 1001 for the Treatment of DM1
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October 19, 2021
Dicerna Reports Mixed Results for Study of Nedosiran in Primary Hyperoxaluria Type 3
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October 19, 2021
Pharnext Reports Positive Results from Late-Stage CMT Trial Published in Journal
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October 18, 2021
LogicBio Reports Data from First In Vivo Genome Editing Therapy in Kids
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October 18, 2021
Biogen ALS Drug Misses Endpoint in Late-Stage Study
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October 18, 2021
FDA Tells Omeros It Will Not Approve Therapy to Treat Side Effect of Stem Cell Transplants
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October 18, 2021
AI Drug Discovery Platform Quris Unveils Itself with $9 million Seed Round
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October 14, 2021
Cyprium Reports Positive Data for CUTX-101 in Pivotal Studies in Menkes Disease
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October 13, 2021
Albireo Enters Exclusive Licensing Agreement with Jadeite Medicines for Bylvay in Japan
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October 12, 2021
Sarepta Therapeutics’ Gene Therapy Shows Sustained Functional Improvements in Patients with DMD
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October 12, 2021
FDA Grants Fast Track Designation to GenSight for Optogenetic Therapy to Treat Retinitis Pigmentosa
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