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- Novartis' del-desiran fails primary endpoint in phase 3 Harbor trial for myotonic dystrophy type 1Novartis announced that its experimental therapy del-desiran did not meet the primary endpoint in the phase 3 Harbor trial, which evaluated improvement in video hand opening time in patients with m…
- Beam Therapeutics reports one-year data from early-stage AATD gene therapy trialBeam Therapeutics released new results from its early-stage gene-editing program targeting alpha-1 antitrypsin deficiency (AATD), a rare condition that can damage the lungs and liver. The data com…