Three of 4 patients withdrew from their previous enzyme replacement therapy following treatment with AT845 gene therapy.
Noah Stansfield
Advertisement
Noah Stansfield, Assistant Editor for NeurologyLive® and CGTLive®, has been with MJH Life Sciences since June 2022. Follow him on LinkedIn or email him at [email protected]
Articles by Noah Stansfield
Descartes-08, an investigational autologous RNA-engineered chimeric antigen receptor T-cell therapy (rCAR-T) has been dosed with the first participant in phase 2b randomized controlled trial.
Recruitment has begun for REGENXBIO's AFFINITY BEYOND clinical trial assessing the prevalence of AAV8 antibodies in male patients with Duchenne muscular dystrophy.
In addition to meeting primary end point of change in North Star Ambulatory Assessment, treatment with SRP-9001 resulted in statistically significant improvements in timed function tests.
Advertisement
Advertisement
Advertisement
Trending on NeurologyLive - Clinical Neurology News and Neurology Expert Insights
1
From Diagnosis to Treatment: Key Questions in Immune-Mediated Small Fiber Neuropathy
2
Current Treatment Strategies for Dravet Syndrome
3
NeurologyLive®'s Conference Coverage, Oveporexton Displays Symptom Improvement in NT1, FDA Allows BLA for AMT-130 in HD
4
Beyond Motor Symptoms: The Power of Connection in Parkinson Disease
5