Neuromuscular

Clinical data on the investigational therapeutics, DNL343 and SAR443820/DNL788, were presented at the 2021 Annual Northeast ALS Meeting this month.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending October 8, 2021.

The organization called attention to unintended harms of the current Medicare benefit structure and the need for the Center for Medicare & Medicaid Services to be more “patient-focused” in its assessments.

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.

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In preclinical mouse models lacking both dystrophin and utrophin, treatment with an agent using the company's PN chemistry resulted in 100% survival at 40 weeks, setting up the therapy for this phase 1b2/a trial.

Findings from studies of the neuromuscular treatments were presented by at the 2021 World Muscle Society Virtual Congress, September 20-24.

Neurology News Network for the week of October 2, 2021. [WATCH TIME: 4 minutes]

Patients with Duchenne muscular dystrophy receiving the treatment had a decline in Mid-level Performance of Upper Limb scores in the phase 2 trial of the cell therapy.

The investigator at the Healey & AMG Center for ALS at Massachusetts General Hospital spoke to the data backing the investigational AMX0035 in the treatment of ALS. [WATCH TIME: 3 minutes]

Expert clinicians offer their perspectives on the ongoing shortage of neurologists, transgender and gender-diverse individuals with migraine, artificial intelligence and neuroimaging, Student Sleep Health Week 2021, and the importance of rehabilitation therapy in multiple sclerosis.

Even after excluding those who received ALS diagnosis in the 5 years prior, higher HDL and APOA1 levels were associated with a lower risk of ALS.

Commenting on recent advances and persistent challenges for these patients, the associate professor of clinical occupational therapy at USC Keck Hospital spoke on the need to increase equity and access to care. [WATCH TIME: 6 minutes]

The agent had previously demonstrated positive results in a phase 2a setting, with favorable trends on modified Unified MSA Rating Scale scores across a 12-week period.

The associate professor of clinical occupational therapy at USC Keck Hospital discussed the role of occupational therapists and other medical professionals in the care of these patient populations. [WATCH TIME: 3 minutes]

The first-of-its-kind, peer-nominated recognition program will celebrate the comprehensive care community in multiple sclerosis.

The associate professor of clinical occupational therapy at USC Keck Hospital discussed the growing awareness for the care of these patients, as well as the integration of occupational therapists. [WATCH TIME: 2 minutes]

With AMX0035 expected to be submitted to the FDA for review in the coming months, the investigator at the Healey & AMG Center for ALS at Massachusetts General Hospital chimed in to share her thoughts on its potential. [WATCH TIME: 3 minutes]

Jess Holguin, OTD, OT/L, spoke on the unique role occupational therapists can play for patients with neuromuscular and movement disorders.

Data from the STRIDE patient registry showed that treatment was also associated with a delay in pulmonary function decline for those with Duchenne muscular dystrophy.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending September 24, 2021.

The expanded access program is designed to provide individuals with ALS who are not eligible to enroll in the Healey ALS Platform Trial access to CNM-Au8.

The director of IT and Neuroinformatics Development at the Buffalo Neuroimaging Analysis Center discussed future neuroimaging projects the center is evaluating and where the focus turns to next. [WATCH TIME: 3 minutes]

Both the 200-mg and 350-mg dose groups showed numerically advantageous differences over placebo in secondary end points such as average daily pain score and 30% reduction in ADP.

Here's what is coming soon to NeurologyLive.