Matt Hoffman

Topline data from the phase 3 ADAPT-SC study (NCT04735432) suggest that the subcutaneous formulation of the Argenx product was statistically noninferior to the recently approved IV formulation, efgartigimod afla-fcab (Vyvgart).

The director of neuromuscular clinical trials at Columbia University Irving Medical Center shared her perspective on the discussions that took place at MDA 2022 and the importance of collaboration among clinicians and researchers.

Data from the ProTEct-MS trial (NCT04480307) suggest that temelimab is safe and well-tolerated in patients with relapsing MS also treated with rituximab, and data were consistent with prior findings. More analysis will be necessary to define an optimal dose.

The gene therapy from Novartis showed that it was both efficacious and well-tolerated in presymptomatic patients with SMA with 3 copies of SMN2, with no patients experiencing treatment-related serious adverse events.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Richard K. Bogan, MD, FCCP, FAASM. [LISTEN TIME: 18 minutes]

Jinsy Andrews, MD, MSc, FAAN, offered her perspective on the ongoing therapeutic boom in neuromuscular disease and some of the critical needs for physicians to keep up with a rapidly progressing treatment paradigm.

Steven Wolf, MD, and Patricia McGoldrick, NP, MPA, MSN, offered their perspectives into the value of awareness days for rare disease communities and the potential to improve the diagnosis of LGS.

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.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Seema Khosla, MD, FCCP, FAASM. [LISTEN TIME: 29 minutes]

The neurodegeneration program lead at Genentech offered his perspective on the phase 3 SKYLINE trial and the historical data on gantenerumab ahead of this new clinical assessment.

The trial will use PACC-5 score as its primary end point and expects to enroll 1200 participants who have confirmed amyloid pathology without signs of cognitive impairment.

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.

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.

Mechanistic data from the phase 2 REPAIR-MS trial support blinded efficacy data from the VISIONARY-MS trial, suggesting that Clene Nanomedicine’s investigational CNM-Au8 has the potential to drive clinically meaningful improvements in recognized MS functional end points.

A pair of datasets from the CLASSIC-MS and GLIMPSE studies of cladribine (Mavenclad; EMD Serono) suggest that the oral tablet sustained delayed conversion to clinically definite MS as well as favorable efficacy to dimethyl fumarate, fingolimod, and teriflunomide.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Konstantin Karmazin, MD. [LISTEN TIME: 26 minutes]

ACTRIMS president Jeffrey Cohen, MD, offered insight into what’s to come from the upcoming Americas Committee for Treatment and Research in Multiple Sclerosis Annual Forum 2022.

Expert neurologist and sleep specialist Chris Winter, MD, provides a practical outlook on the identification and management of narcolepsy and excessive daytime sleepiness.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Dhanashri P. Miskin, MD. [LISTEN TIME: 39 minutes]

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.

Catch up on any of the neurology news headlines you may have missed over the course of the last month, compiled all into one place by the NeurologyLive® team.

Expert clinicians offer their insight on conducting trials in pediatric migraine, the pipeline of Alzheimer therapies, disorders of consciousness and COVID-19, and more.

After a prior phase 2 failure in CHANGE-MS showed signs of promise for progressive disease, the final patient visit has been completed in a trial of temelimab in relapsing disease, with results expected before the end of March 2022.

After discontinuing dosing in the phase 3 GENERATION HD1 study in 2021, Roche is designing a new phase 2 trial for tominersen, its investigational antisense oligomer for Huntington disease.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Bridget A. Bagert, MD, MPH. [LISTEN TIME: 32 minutes]

The senior VP of clinical research and deputy chief clinical officer of the neurology business group at Eisai spoke to the recent first enrollment in the Tau NexGen study of the investigational antimicrotubule binding region tau antibody, E2814.

After a post hoc analysis revealed promising results in a subgroup of younger adults with less disease burden, Ionis has announced that Roche will develop a new phase 2 trial.

Following the publication of data suggesting that EBV is a leading cause of MS, the director of the Ochsner MS Center and an investigator in an EBV-specific therapy clinical trial offered her reaction to the data.

Becky Tilahun, PhD, a clinical psychologist at Cleveland Clinic, offered insight into a recent study she and colleagues conducted exploring CBT-informed psychotherapy for patients with psychogenic nonepileptic seizures

Expert clinicians offer their insight on costs of Alzheimer drug development, a new agent for generalized myasthenia gravis, migraine in the emergency department, educational sleep medicine “boot camps”, AES 2021, and more.