Neuromuscular

Over a 24-week period, DNL343 failed to distinguish itself from placebo on the primary end point of ALSFRS-R, as well as on other secondary end points of muscle strength and respiratory function.

Most participants showed improvements in motor function, with 61% achieving a clinically meaningful increase in HFMSE scores by week 52 and 41% achieving similar gains in RULM scores.

Catch up on any of the neurology news headlines you may have missed over the course of November 2024, compiled all into one place by the NeurologyLive® team.

As part of NeurologyLive®'s Year in Review, we've compiled the most-watched expert-led video series that appeared on the website in 2024.

David Lynch, MD, PhD, a professor of neurology at the University of Pennsylvania Perelman School of Medicine, discussed the significance of a number of recently presented analyses highlighting omaveloxlone, the first approved therapy for Friedreich ataxia.

Imlifidase-IVIg treatment led to faster disability improvement (3 weeks) and earlier independent walking (6 weeks) compared to IVIg alone.

Novartis plans to present OAV101 IT data at a 2025 medical meeting and share results with regulatory agencies, including the FDA, to support approval for patients with SMA in need.

Capricor may qualify for a priority review voucher from the FDA if deramiocel receives marketing approval for the treatment of Duchenne muscular dystrophy.

As part of NeurologyLive®'s Year in Review, we've compiled the most-listened episodes of our biweekly podcast, Mind Moments®.

As part of NeurologyLive®'s Year in Review, we've compiled the most-watched interview clips that appeared on the website in 2024.

As part of NeurologyLive®'s Year in Review, take a look at our most-read news in neuromuscular disorders in 2024.

As part of NeurologyLive®'s Year in Review, we've compiled the top coverage stories from 2024 across a variety of different neurological conferences the team either attended or covered virtually.

As part of NeurologyLive®'s Year in Review, we've compiled the most-read feature-length stories that appeared on the website in 2024.

The complement system is critical in immune defense and tissue homeostasis, but its dysregulation can contribute to autoimmune neurological disorders and neurodegenerative diseases like Alzheimer, ALS, and multiple sclerosis.

Vatiquinone, a small molecule inhibitor, demonstrated its efficacy and safety across a range of age groups with Friedreich ataxia, with effects seen on disease progression and specific subscales of the mFARS.

Human studies revealed thinner myelin sheaths in CD59-deficient patients, indicative of a process of segmental demyelination followed by remyelination.

Managing ALS, particularly its respiratory complications, requires a collaborative multidisciplinary team—including neurologists, pulmonologists, and respiratory therapists—working together to address the complexities of respiratory failure, the leading cause of death in these patients.

The program assembles a collaborative team of in-house and external experts in research, clinical care, and regulatory affairs to navigate gene therapy programs through the drug development process.

The proof-of-mechanism study will evaluate single doses of QRL-101 in approximately 12 patients with ALS, focusing on excitability biomarkers like the strength-duration time constant (SDTC), safety, tolerability, and plasma pharmacokinetics, with topline results expected in early 2025.

As of July 2024, 2 participants in the ongoing CONNECT1 study have received 4 doses of PGN-EDO51 at 10 mg/kg, which has been generally well tolerated, with initial results expected in early 2025.

The positive data, along with data from GRAND CANYON anticipated in early 2025, are thought to be part of a potential future authorization submission for sevasemten in Becker.

Donald S. Wood, PhD, president and chief executive officer at the MDA, provided thoughts and perspectives on the promising design and educational attainment for the 2025 MDA Clinical & Scientific conference.

The FDA recommended a follow-up meeting with Clene to further discuss the analyses needed to support the accelerated approval pathway for CNM-Au8 in amyotrophic lateral sclerosis.

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

RGX-202 demonstrated improved functional outcomes and robust microdystrophin expression, with levels exceeding those of other approved or investigational gene therapies.

Perspective from neurologists on the current and future applications for artificial intelligence across a bevy of neurologic conditions.

A recently initiated phase 1/2 trial of NS-050/NCNP-03 will assess patients with Duchenne muscular dystrophy on dystrophin production, muscle strength, mobility, and functional exercise capacity.

Catch up on any of the neurology news headlines you may have missed over the course of November 2024, compiled all into one place by the NeurologyLive® team.

Rachana K. Gandhi Mehta, MBBS, an assistant professor of neurology at Wake Forest School of Medicine, discussed a new, innovative trial assessing the therapeutic potential of self-administered subcutaneous rozanolixizumab as a treatment for myasthenia gravis.

QRL-201, developed by QurAlis, aims to restore STATHMIN-2 (STMN2) expression, a key protein for neural repair, which is significantly reduced in patients with ALS.