Neuromuscular

The adjunct professor of human genetics at Emory University School of Medicine discussed previous research on Rett syndrome that initially provided crucial insight on the neuromuscular condition in adult populations. [WATCH TIME: 2 minutes]

Despite conditional approval in Europe since 2014, ataluren's authorization has faced rejections and is still investigational in the US.

VCA-894A, a novel antisense oligonucleotide, exhibits a mechanism of action that specifically targets a cryptic splice site variant within immunoglobulin mu-binding protein 2.

Despite initial enthusiasm, novel treatments for Duchenne muscular dystrophy face discontinuation challenges and lack long-term clinical efficacy data.

Over a 28-week treatment period, the investigational agent demonstrated significantly greater increase in dystrophin expression and exon skipping than previously approved eteplirsen, Sarepta’s first therapy for Duchenne muscular dystrophy.

Take a look at some of the most-anticipated FDA pending approvals expected in 2024 that researchers and clinicians in neurology should keep an eye out on.

Takeda's FDA-approved Gammagard Liquid can be used as induction therapy, which includes an induction dose followed by maintenance doses, for adults with chronic inflammatory demyelinating polyneuropathy.

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

The chief executive officer of ClearPoint Neuro discussed how the company’s SmartFrame OR Stereotactic System can facilitate neurosurgery and the next steps in integrating it into clinical settings. [WATCH TIME: 4 minutes]

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 movement disorders.

Even after adjusting for indications for benzodiazepine administration and baseline disease severity, results showed greater patient disease worsening while on such medications, regardless of duration and dose.

A systematic approach to understanding the needs of the caregiving youth—a growing and less-studied population of caregivers—is needed to better tailor interventions, provide support, and improve care to patients and their families.

Neurology News Network for the week ending January 27, 2024. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending January 26, 2024.

In total, 82% of patients were interested in more convenient treatments, emphasizing the importance of shared decision-making and open discussions with physicians in rare disease management.

The investigational ALS therapy, IPL344, derived from a biologically active peptide, exhibited encouraging efficacy in slowing disease progression.

The professor of neurology at Cedars-Sinai Medical Center shared his thoughts on potential therapies that expand management care options for patients with neuromuscular disease, in particularly for chronic inflammatory demyelinating polyneuropathy. [WATCH TIME: 4 minutes]

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

The professor of neurology at Cedars-Sinai Medical Center shared his clinical perspective on the recent FDA approval of Takeda’s immune globulin infusion, Hyqvia, for chronic inflammatory demyelinating polyneuropathy. [WATCH TIME: 5 minutes]

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 headache and migraine.

Neurology News Network for the week ending January 21, 2024. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending January 19, 2024.

PARADIGM, an ongoing phase 2 placebo-controlled trial, will have additional biomarker data released in the first half of 2024.

Aruna Bio's investigational treatment, AB126, is now set for a phase 1b/2a clinical trial targeting acute ischemic stroke, anticipated to begin in the first half of 2024.

Takeda's Hyqvia is the only FDA-approved combination of immunoglobulin and hyaluronidase, which makes it a facilitated subcutaneous immunoglobulin infusion, for patients with chronic inflammatory demyelinating polyneuropathy.

Despite lack of efficacy in the phase 3 ADORE trial, the oral formulation of edaravone demonstrated safety and tolerability during a 48-week daily dosing period.

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 epilepsy and seizure disorders.

The pediatric REVEAL trial assesses THSA-102 in 2 cohorts of pediatric patients with Rett syndrome due to MECP2 loss-of-function mutation, with results expected in mid-2024.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending January 12, 2024.

The study investigates the potential benefits of ThecaFlex DRx System, an implantable intrathecal catheter, for patients with SMA treated with nusinersen, aiming to reduce the need for serial lumbar punctures and enhance the overall administration experience.