Neurology

SRP-9003 elicited sustained protein expression in muscle tissue and stabilized North Star Assessment for Dysferlinopathies scores at 2 years in patients with limb-girdle muscular dystrophy Type 2E.

Updated findings from across a number of studies highlighted the significant benefits of onasemnogene abeparvovec for children with spinal muscular atrophy.

Ninety percent of patients with cerebral adrenoleukodystrophy who were treated with elivaldogene autotemcel were alive and free of major functional disabilities at 2-years.

The FDA has granted a fast track designation to PBFT02, PBKR03, and PBGM01, which are intended as treatments for patients with various rare CNS disorders.

Orchard Therapeutics released its plans for 2021, which included the launch of OTL-200 (Libmeldy) in Europe and development of an FDA submission strategy in the United States.

Rocket Pharmaceuticals has summarized multiple major milestones for its 5 gene therapies from 2020 and provided a forecast and outlook for what is expected to be a banner year for the organization.

Advancements in gene therapy approaches have opened the door for a glimpse into the future of care for a number of diseases.

FDA advises against submission of NurOwn as a treatment for ALS, based on a lack of statistical significance in phase 3 study.

Masahito Kawabori, MD, PhD, associate professor, Hokkaido University, discussed results of the phase 2 STEMTRA trial.

Vamshi Rao, MD, attending physician of Neurology at Ann and Robert H. Lurie Children’s Hospital of Chicago, discusses the safety and efficacy of spinal muscular atrophy (SMA) treatments.

Descriptive findings from the phase 1/2 study allude to promise for the gene therapy in the rare pediatric disorder.

New phase 2/3 findings meet investigators' hypotheses on the gene therapy's mixed benefits for heparan sulfate reduction.

Investigators observed sustained, dose-dependent benefits for young patients at 24 months.

Vamshi Rao, MD, attending physician of Neurology at Ann and Robert H. Lurie Children’s Hospital of Chicago, discusses how disease modifying therapies have improved spinal muscular atrophy (SMA) prognosis.

The associate professor at Hokkaido University and investigator of STEMTRA also discussed other efforts in stroke, Alzheimer disease, and Parkinson disease.

The Pfizer candidate PF-06939926 has also received fast track, orphan drug, and rare pediatric disease designations from the FDA.

Minimal fat infiltration was observed on MR images from the SRP-9001 arm compared to participants from the natural history cohort.

Results of the phase 2 study support continued dose escalation of SRP-5051 and further clinical development.

These data follow a recent announcement from Sio Gene Therapies that the first patient in the high-dose cohort has been dosed.

Solid Biosciences noted that its response to the agency requests for further information about manufacturing as well as updated safety and efficacy data on all dosed patients led to the lift.

Nearly all patients in this cohort achieved a clinically meaningful >3-point increase during the study period, demonstrating a consistent response to the gene therapy.

The agency said the gene therapy should remain on the market while it assesses the situation and does not impact their evaluation of data from the human clinical trials.

Interim data from the first 8 pediatric patients showed that the AAV-CLN6 gene therapy demonstrated a positive impact on motor and language function compared to a natural history dataset, as well as in comparison to in-study sibling pairs.

Preclinical trials and success stories suggest that much is riding on vector-based therapies for the treatment of rare neurological conditions.

AveXis—a Novartis company—announced that it will work with payers to implement 5-year outcomes-based agreements and novel pay-over-time options. The company also said it will offer a patient program to support affordability and access.