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In preclinical studies, ENTR-601-44 has demonstrated a robust ability to skip exon 44 in the muscles of nonhuman primates and full-length human DMD mice.

After 6 weeks of treatment, WVE-N531 was found to be safe, well-tolerated, and resulted in mean dystrophin production that was below the level of quantification.

Here's some of what is coming soon to NeurologyLive® this week.

Neurology News Network for the week ending December 17, 2022. [WATCH TIME: 4 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending December 16, 2022.

AOC 1001, an agent consisting of a proprietary monoclonal antibody that binds to the transferrin receptor 1, was safe, tolerable, and showed significant reductions in DMPK, a disease-related mRNA.

In a phase 3 trial, NurOwn failed to meet its primary end point of change on ALSFRS-R; however, the therapy showed significant benefits in those with less severe forms of ALS.

Catch up on any of the neurology news headlines you may have missed over the course of the last month, compiled all into one place by the NeurologyLive® team.

Here's some of what is coming soon to NeurologyLive® this week.

High rates of treatment use were observed across all types of spinal muscular atrophy, with nusinersen, the first approved disease-modifying treatment, as the most commonly used.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is Alzheimer disease and related dementias.

Neurology News Network for the week ending December 10, 2022. [WATCH TIME: 4 minutes]

As the treatment paradigm for neurologic diseases rapidly progresses, the need for more thorough biomarker tools to measure disease progression and severity has increased, and in recent years, GFAP has emerged as a valuable candidate to add to the existing panel.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending December 9, 2022.

After showing robust inhibition of biomarkers associated with integrated stress response, the eIF28 modulator will be assessed alongside several other potential agents in the HEALEY ALS Platform trial.

Here's some of what is coming soon to NeurologyLive® this week.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is the history of the American Epilepsy Society.

Neurology News Network for the week ending December 3, 2022. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending December 2, 2022.

The president and CEO, as well as the executive vice president and chief research officer, of MDA, together offered an in-depth overview of what to expect from the 2023 MDA Conference, which will be held from March 19-22, 2023, in Dallas, Texas. [WATCH TIME: 12 minutes]

The PDUFA date for SRP-9001, developed in partnership between Sarepta and Roche, is set for May 29, 2023. The BLA was submitted for accelerated approval.

The division chief of stroke and vascular neurology at Duke Health provided perspective on the multidisciplinary team needed to treat neuromuscular poststroke symptoms, as well as exciting innovations on the horizon. [WATCH TIME: 4 minutes]

The president of the International Neuropalliative Care Society detailed potential disease states that could benefit from neuropalliative care as well as the importance of the patient community in expanding this type of approach. [WATCH TIME: 4 minutes]

Here's some of what is coming soon to NeurologyLive® this week.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is sleep disorders.



























