Neuromuscular

At each timepoint, all assessed participants with 3 SMN2 copies and the majority with 2 SMN2 copies were identified by PASA as not being tube fed.

Treatment with nusinersen demonstrated no clinically meaningful changes in vital signs or clinical laboratory parameters, including urine parameters, in those with spinal muscular atrophy.

Researchers also found that, in comparing ataluren to standard care, ataluren delayed loss in pulmonary function.

The high-dose cohort in part 1 of the FIREFISH study also showed that more than 50% of infants could sit without support and more than 90% could feed orally.

Here's what is coming soon to NeurologyLive.

Students share their experience in the virtual classroom, navigating new requirements, and the lessons learned from their position in a global pandemic.

Sharon Hesterlee, PhD, executive vice president and chief research officer, MDA, shares an early look at the MDA's 2021 Virtual Clincial and Scientific Conference, March 15-18.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending March 12, 2021.

The executive vice president and chief research officer of the MDA gave an overview of the upcoming virtual annual meeting and her insight on what to look for.

Researchers found that the proportion of patients meeting DNC criteria suitable for organ donation increased after teleneurology use.

Garey H. Noritz, MD, walks us through a virtual pediatric neurological exam and notes examination findings that may be identified in an infant with Spinal Muscular Atrophy (SMA).

Diana Castro, MD walks us through a pediatric neurological exam and notes examination findings that may be identified in an infant with Spinal Muscular Atrophy (SMA).

Treatment with masitinib was shown to prevent the amyloid-induced hemichannel-dependent mast cell activity in bone marrow-derived mast cells and brain mast cells.

The director of research analytics at Cure SMA detailed the ways to decrease care management costs for patients with spinal muscular atrophy and the role incoming treatments can play.

Here's what is coming soon to NeurologyLive.

The 2-stage study is an open-label, 2-stage clinical trial designed to evaluate safety and dose-escalation (stage 1) and safety and efficacy (stage 2) of surgically delivered AXO-AAV-GM2.

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

The director of research analytics at Cure SMA discussed her research on the economic burden and costs patients with spinal muscular atrophy and their caregivers face.

Protalix BioTherapeutics intends to report final data on the agent, also known as PRX-102 in the second half of 2021, as well as present those findings at an appropriate medical conference.

The FIREFISH clinical trial by PTC Therapeutics is 1 of several studies underway evaluating risdiplam in SMA.

The recommendation stems from recently published topline data which demonstrated a clinically meaningful treatment response compared to placebo but failed to meet statistical significance.

Here's what is coming soon to NeurologyLive.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending February 26, 2021.

The antisense oligonucleotide casimersen will be branded as Amondys 45 and is currently being assessed in the ESSENCE phase 3 clinical trial (NCT02500381).

Advancements in gene therapy approaches have opened the door for a glimpse into the future of care for a number of diseases.