Neuromuscular

The expert panel comments on ideal patient profiles for SMA treatment with nusinersen, onasemnogene abeparvovec, and risdiplam. O painel de especialistas comenta os perfis ideais de pacientes para tratamento da atrofia muscular espinhal com nusinersena, onasemnogeno abeparvoveque e risdiplam.

Drs Rodrigo de Holanda Mendonça and Adriana Banzzatto Ortega discuss clinical trial data with nusinersen, onasemnogene abeparvovec, and risdiplam. Drs Rodrigo de Holanda Mendonça e Adriana Banzzatto Ortega discutem dados de testes clínicos com nusinersena, onasemnogeno abeparvoveque e risdiplam.

Recruitment has begun for REGENXBIO's AFFINITY BEYOND clinical trial assessing the prevalence of AAV8 antibodies in male patients with Duchenne muscular dystrophy.

An exergaming program that spanned 8 weeks showed positive effects for patients with multiple sclerosis both with and without restless legs syndrome.

With a PDUFA date of October 26, 2023, the FDA will review data from the phase 2b VISION-DMD study, in which vamorolone met its primary end point in change in TTSTAND velocity.

Take a look at 5 of the most-anticipated clinical trial data readouts expected in 2023 that neurology health care professionals should keep their eyes on.

The executive direct of Cure CMD discussed the state of care for patients with congenital muscular dystrophies, the need for new treatments, and the patience with gene therapies.

Here's some of what is coming soon to NeurologyLive® this week.

Richard Williamson, MD, FAANS, a neurosurgeon at Allegheny Health Network, provided commentary on the explosion of technology in neurosurgery and how exoscopes ease the process.

The executive director of Cure CMD discussed efforts being done to bring the stakeholder community together and improve the care for patients with congenital muscular dystrophies. [WATCH TIME: 4 minutes]

Neurology News Network for the week ending January 28, 2023. [WATCH TIME: 4 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending January 27, 2023.

Take a look at 5 of the most-anticipated FDA pending approvals expected in 2023 that neurological researchers and clinicians should keep an eye out on.

Drs Juliana Gurgel Giannetti and Rodrigo de Holanda Mendonça provide an overview of available treatments for spinal muscular atrophy in Brazil. / Drs Juliana Gurgel Giannetti and Rodrigo de Holanda Mendonça discutem tratamentos para atrofia muscular espinhal disponíveis no Brasil.

Drs Juliana Gurgel Giannetti and Rodrigo de Holanda Mendonça discuss genetic changes in spinal muscular atrophy and genetic screening in Brazil. / Drs Juliana Gurgel Giannetti and Rodrigo de Holanda Mendonça discutem mudanças genéticas em atrofia muscular espinhal e screening genético no Brasil.

The Abbott system previously earned approval for chronic pain in 2019, and adds another option for a patient population with no disease-modifying agents available for treatment.

Drs Juliana Gurgel Giannetti, Adriana Banzzatto Ortega and Marcelo Kerstenetzky discuss symptoms of spinal muscular atrophy and diagnosis and referral process in Brazil. / Drs Juliana Gurgel Giannetti, Adriana Banzzatto Ortega e Marcelo Kerstenetzky discutem sintomas da atrofia muscular espinhal, diagnóstico e processo de encaminhamento no Brasil.

Dr Rodrigo de Holanda Mendonça provides an overview of spinal muscular atrophy (SMA). / Dr Rodrigo de Holanda Mendonça dá uma visão geral sobre Atrofia Muscular Espinhal (AME).

Ahead of the 2023 MDA Conference, which will be held from March 19-22, 2023, in Dallas, Texas, the vice president of public policy and advocacy at the Muscular Dystrophy Association provided an overview of the current state of access and the need for more advocates in neuromuscular disease. [WATCH TIME: 14 minutes]

Similar to results at 24-months, findings showed improvements in patient outcomes with the SCS System including quality of life, functional ability, mood, and sleep.

Here's some of what is coming soon to NeurologyLive® this week.

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 genetic neurological disorders.

After a 12-month period, patients were able to use the implant for routine digital activities, such as texting, emailing, personal finance, online shopping, and communication of care needs.

The FDA lifted its clinical hold on Astellas Pharma's FORTIS trial for evaluating AT845 in adults with late-onset Pompe disease.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending January 20, 2023.