Neuromuscular

The global compound development team leader at Janssen discussed the importance of Rare Disease Day and one in particular, myasthenia gravis, which affects approximately 36,000 to 60,000 people in the US. [WATCH TIME: 3 minutes]

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Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending February 25, 2022.

Despite the majority of program directors reporting the importance of training in cerebral palsy, they also felt that residents were not very well prepared to manage the condition

Tahseen Mozaffar, MD, FAAN, spoke on recently reported positive interim safety data from the phase 1/2 FORTIS study, noting that the treatment was thus far safe and well-tolerated in patients.

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Although pain is a known problem for children and adolescents with the disease, not much is understood about pain in adults with CP.

In contrast to general turning measures and the Scale for the Assessment and Rating of Ataxia score, lateral velocity change allowed detecting longitudinal changes in 1-year follow-up recordings.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending February 18, 2022.

The director of Cleveland Clinic’s Epilepsy Center discussed the currently known overlap between neurological diseases and the need to identify causative biomarkers. [WATCH TIME: 4 minutes]

With the physician deficit projected to grow larger within a decade, this global challenge has become a major focus of large organizations and medical societies.

The director of the Division of Neuromuscular Diseases at UC Irvine discussed the positive data, noting that the treatment was “incredibly safe.” [WATCH TIME: 4 minutes]

Advances in technology and research have pushed the field to the brink of a revolutionary era of treatment.

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Although we have entered the age of genetically targeted therapies in the neuromuscular clinic, there are many unresolved clinical, economic, and ethical questions that require extensive further research.

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

The director of Cleveland Clinic’s Epilepsy Center provided insight on a new 20-year initiative to uncover more about the origins of neurological diseases and how they occur prior to symptom onset. [WATCH TIME: 3 minutes]

The director of Cleveland Clinic’s Epilepsy Center discussed the new Cleveland Clinic Brain Study, which will evaluate predisease fingerprints from patients who go on to develop neurological disorders.

FORTIS (NCT04174105) is a first-in-human study, enrolling 4 participants with late-onset Pompe disease to assess the Astellas Pharma therapy.

Here's what is coming soon to NeurologyLive®.

After changes were made to the AVATAR phase 3 study in Rett syndrome, Anavex provided context on the timing of the adjustments, which were approved by regulatory bodies in the UK and Australia, where the trial was conducted.

Matthew Klein, MD, MS, FACS, the COO of PTC Therapeutics, discussed a supplemental new drug application for the treatment in a younger group of patients, which has been granted priority review by the FDA.

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

The biotech company announced a collaboration with NS Pharma around CAP-1002’s commercialization and distribution ahead of its HOPE-3 phase 3 trial in Duchenne muscular dystrophy.

In the RAISE trial, patients treated with zilucoplan exhibited clinically meaningful and statistically significant improvement from baseline in Myasthenia Gravis-Activities of Daily Living Profile total score at week 12, when compared with those who received placebo.

The assistant clinical professor at Vanderbilt University School of Medicine discussed ways of incorporating and encouraging women physicians to enter the neurology specialty. [WATCH TIME: 4 minutes]

Most child neurologist respondents were generally early in their career, had diverse subspeciality training, and tended to work at large, resource-rich centers capable of multimodal neuromonitoring.

Although research illustrates the persistence of inequities between women and men in neurology, 3 women leaders shared encouraging thoughts and experiences highlighting the progress made in recent years.

Collaboration can result in greater success than what one can achieve alone, and for women physicians, this rings truer than ever before, writes Jill M. Farmer, DO, MPH.

In light of National Women Physicians Day, the assistant clinical professor at Vanderbilt University School of Medicine commented on the roles women have played historically, and her perspective on where they stand now. [WATCH TIME: 12 minutes]