Neuromuscular

Data may be useful when analyzing disease progression and treatment efficacy in future facioscapulohumeral muscular dystrophy clinical trials.

The treatment was well tolerated at 48 weeks in patients with DMD with a confirmed mutation amenable to exon 45 skipping.

Part B of the 2-part study will have patients previously on placebo receive SRP-9001 for an additional 52 weeks.

The 2022 MDA National Ambassador and patient with Bethlem myopathy spoke to the importance of including the patient perspective at medical meetings to improve the care paradigm in neuromuscular disorders. [WATCH TIME: 3 minutes]

Individuals on ravulizumab demonstrated statistically significant changes in the primary end point of MG-ADL and in secondary end points such as Quantitative Myasthenia Gravis total score.

At 97 weeks, avalglucosidase alfa showed continued benefit on measures such as forced vital capacity and 6-minute walk test distance and was successful for patients who switched off algucosidase alfa.

Data from the phase 2 NURTURE study of nusinersen (Spinraza; Biogen) suggest that long-term treatment with the agent is beneficial, and point to the importance of newborn screening and early treatment for spinal muscular atrophy.

Here's what is coming soon to NeurologyLive®.

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 sleep disorders.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending March 11, 2022.

The Muscular Dystrophy Association's annual meeting will take place in-person and virtually in Nashville, Tennessee, on March 13-16, with more than 950 in-person attendees and 130 presenters.

In the EVOKE study (NCT02924129), the spinal cord stimulation system demonstrated superior pain relief compared to open-loop SCS at 12 months with no increase in pain medication.

The assistant professor of pediatric neurology at Washington University in St. Louis provided an overview of cerebral palsy and ways to reduce the risk of developing the condition. [WATCH TIME: 3 minutes]

Here's what is coming soon to NeurologyLive®.

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 nutrition in multiple sclerosis.

Neurology News Network for the week ending March 4, 2022. [WATCH TIME: 4 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending March 4, 2022.

Crystal Proud, MD, demonstrates a typical neurological telehealth exam for pediatric SMA with one of her patients.

Families with patients with SMA share advice for community neurologists on helping new patients through the diagnostic process.

Patients describe how their lives have changed since their SMA diagnosis, and a neurologist provides advice for community physicians.

Crystal Proud, MD, and Craig Zaidman, MD, discuss the necessity and urgency of genetic testing for SMA in children and adolescents.

Experts explain the next steps for families and physicians after SMA is suspected in a child.

An overview of the signs and symptoms that physicians look for in telehealth exams when SMA is suspected.

In a pilot study in late-onset Pompe disease, patients demonstrated improvements in leg extensor, flexor strength, and maximum inspiratory pressure following resistance training.

A discussion of the benefits of using telehealth for SMA management for clinicians and families.

Two pediatric neurologists describe their experiences with telehealth appointments for SMA during the COVID-19 pandemic.

Craig Zaidman, MD, discusses how critically important newborn SMA screening is and early diagnosis and treatment of SMA.

Families describe their diagnostic journeys with SMA and when they were referred to SMA specialists.

Dr Craig Zaidman details the symptoms a physician looks for in a pediatric patient when making an SMA diagnosis.

Families with patients with SMA explain the signs and symptoms shown by their children that prompted a doctor’s visit.