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Oak Hill Bio announced dosing of the first participant in BEACON, a pivotal phase 3 trial evaluating rugonersen, an antisense oligonucleotide originally developed by Roche, for Angelman syndrome.

Alexandra Bonner, MD, a pediatric neuromuscular neurologist at Cleveland Clinic, discussed which specialties are essential to a spinal muscular atrophy care team and why anticipating future needs has become central to the work.

A recently published case study highlights a 17-year-old patient with Duchenne muscular dystrophy who experienced acute and delayed cardiac toxic effects under delandistrogene moxeparvovec treatment, with tissue analyses showing low-level, heterogeneous microdystrophin expression.

Stacey Tarrant, RD, LDN, discusses how earlier treatment alongside disease-modifying therapies have changed nutritional priorities in spinal muscular atrophy, including the need to optimize growth, strength, function, and body composition.

The new data from ALKIVIA marks the first phase 3 study to show statistically significant, clinically meaningful improvement in immune-mediated necrotizing myopathy, a subtype with no approved therapy.

A phase 3 trial found that solriamfetol significantly improved wakefulness and reduced excessive daytime sleepiness in Chinese patients with obstructive sleep apnea, with a safety profile consistent with prior studies conducted in Western populations.

W. Bryan Burnette, MD, chief of neurology at Nemours Children’s Health in Jacksonville, Florida, discussed why access to adult subspecialty care has become one of the largest remaining challenges in spinal muscular atrophy.

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

A large claims-based cohort study found that two doses of the recombinant zoster vaccine reduced the risk of shingles by 64% to 81% in adults with multiple sclerosis, a population with elevated shingles risk and historically low vaccine uptake.

A large multicenter study found that APOE genotype and a polygenic Alzheimer risk score together predict up to a 10-year difference in disease onset among people carrying an extra copy of the APP gene, whether through an APP duplication or Down syndrome.

Experimental small molecule CN045 promoted oligodendrocyte maturation and modest remyelination in preclinical models of multiple sclerosis, supporting further development of the compound as a potential remyelination therapy.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending August 14, 2026.

Clinician author Kevin Chang, PharmD, writes about PACAP as a distinct, druggable migraine pathway independent of CGRP, offering a promising option for patients who don't respond to current therapies.