Neuromuscular

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending August 6, 2021.

Branded as Nexviazyme, Sanofi’s enzyme replacement therapy demonstrated key improvements in measures of disease burden while maintaining its known safety profile.

James Wymer, MD discusses his firsthand experience with risdiplam and identifies a key unmet need in the treatment of spinal muscular atrophy.

An expert neurologist emphasizes the importance of medication compliance and the value of multidisciplinary management in meeting patient goals of therapy for spinal muscular atrophy.

Arun S. Varadhachary, MD, PhD shares insights into clinicians’ and patients’ experiences with medical therapy, comments on unmet needs for adult patients, and speculates about the future of SMA care.

LX1004 and APB-102 are currently in the early phases of research to treat CLN2 Batten disease and SOD1 amyotrophic lateral sclerosis, respectively.

Here's what is coming soon to NeurologyLive.

Higher percentages of infants were event-free and were classified as having a motor-milestone response at month 12 than in historical cohorts.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending July 30, 2021.

An insight into the goals of therapy and treatment selection between oral and intrathecal medications for adult-onset spinal muscular atrophy.

The label now includes the use of ultrasound as a muscle localization technique in adults with spasticity, and includes muscles of the elbow, forearm, hand, and thumb.

A discussion of the multidisciplinary treatment approach in spinal muscular atrophy.

A specialist in the care of patients with spinal muscular atrophy reviews the CHERISH trial of nusinersen and examines the drug’s safety and efficacy profiles.

A key opinion leader in neurology highlights the value of a multidisciplinary approach to care for patients with spinal muscular atrophy in order to maximize a patient’s function through physical and medical therapy.

Patients with neurological sequelae from COVID-19 were significantly more likely to die before discharge (22.2%) compared with non-neuro-COVID patients (14.4%).

Many applicants referenced an early interest and exposure to neurology, often describing their first courses in neurology sparking their desire to learn more about how the brain works.

Here's what is coming soon to NeurologyLive.

The chief medical officer of Scholar Rock discussed the upcoming phase 3 trial for apitegromab in SMA and whether a successful trial would warrant FDA submission.

An overview of the diagnosis of spinal muscular atrophy, including prevalence, age of onset, and the role of genetic testing.

James Wymer, MD, provides a case overview of a 38-year-old female diagnosed with spinal muscular atrophy.

An expert neurologist, Arun S. Varadhachary, MD, PhD considers the diagnostic process for SMA and emphasizes the importance of early diagnosis to optimize care.

Arun S. Varadhachary, MD, PhD provides an overview of the case of a 28-year-old male with spinal muscular atrophy, describes the 4 main phenotypes of SMA, and considers how average age of onset impacts patient prognosis.

Basis for the approval was the SENZA-PDN randomized controlled trial, which showed the system’s ability to substantially sustain pain relief and improve health-related quality of life.

The chief medical officer of Scholar Rock discussed the greatest areas of concern for patients with SMA and why most of these issues exist in the diagnostic stage.

John Brandsema, MD, and Julie Parsons, MD, review the evolution of combination therapy in clinical practice for the management of SMA.