Neuromuscular

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.

A group of experts in the care of patients with rare diseases—James F. Howard, MD; Sabrina Paganoni, MD, PhD; Darcy Kruger, MD, PhD; Veronica Hood, PhD; and Bruce Cree, MD, PhD—shared their perspectives on hot topics of treatment and management.

On Rare Disease Day, the director of the Division of Neuromuscular Diseases at UC Irvine commented on the potential of AT845 for this patient population. [WATCH TIME: 3 minutes]

In recognition of Rare Disease Day, the NeurologyLive® team offered an extensive update on the state of care and treatment for a wide range of rare neurological diseases, including Kleine-Levin syndrome, amyotrophic lateral sclerosis, cerebral palsy, Lennox-Gastaut syndrome, and more.

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]

Here's what is coming soon to NeurologyLive®.

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.

Here's what is coming soon to NeurologyLive®.

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.

Here's what is coming soon to NeurologyLive®.

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.