Neuromuscular

Alnylam’s investigational subcutaneous RNAi therapy is in development for both wild-type and hereditary transthyretin-mediated amyloidosis, with data from the phase 3 HELIOS-A trial demonstrating its potential in treating associated polyneuropathy.

Following their data presentation on AMX0035 at AAN 2022, Justin Klee and Josh Cohen, the co-CEOs and co-founders of Amylyx Pharmaceuticals, shared their perspectives on the agent.

The investigational agent was well-tolerated, with no new safety signals and no indication of worsening of supine hypertension based on 24-hour monitoring.

Adverse events were balanced across treatment and placebo groups, with no observed treatment-associated elevations in cerebrospinal fluid white blood cell counts or protein.

Shephard Mpofu, MD, the senior vice president and chief medical officer at Novartis Gene Therapies, offered his perspective on the latest phase 3 data of onasemnogene abeparvovec (Zolgensma) in SMA treatment.

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After showing several significant differences between placebo, the company has planned a phase 3 study that will assess the therapeutic in a cohort of 230 adults with FSHD.

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 the history of the American Academy of Neurology.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending April 8, 2022.

Seth M. Keller, MD, chair of the Section for Adults With IDDs at AAN, shared his perspective on the state of care for these individuals and the need to improve the difficulties they face when transitioning out of pediatric care.

In the AAN 2022 presidential plenary session, a trio of neurologists discussed a variety of issues stemming from the COVID-19 pandemic and how they impacted research efforts in neurological diseases.

The professor of neurology at NYU Langone Grossman School of Medicine discussed her talk at AAN 2022 on neurological sequelae and follow-up in hospitalized and nonhospitalized patients with COVID-19. [WATCH TIME: 8 minutes]

After switching from nusinersen to Zolgensma 147 days into life, the infant scored a 64 on CHOP-INTEND score, considerably higher than what is observed in the natural history.

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The Bushell Chair of Neurology at the University of Sydney offered his perspective on phase 2 data of CNM-Au8 in amyotrophic lateral sclerosis and spoke to the advances that the field has made in recent years. [WATCH TIME: 3 minutes]

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 Alzheimer disease and dementia.

Interim data from the phase 2 RESCUE-ALS trial open-label extension suggest that Clene Nanomedicine’s investigational agent, CNM-AU8, offered benefits to patients with ALS in terms of survival, in addition to prior results suggestive of slowed progression.

Neurology News Network for the week ending April 2, 2022. [WATCH TIME: 3 minutes]

The Bushell Chair of Neurology at the University of Sydney shared his insight into the latest data on CNM-Au8 as a potential treatment for amyotrophic lateral sclerosis and the frameshift in treatment that it represents. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending April 1, 2022.

The president and chief executive officer of The ALS Association provided insight on the most pressing needs for patients with ALS and the organization’s focus going forward. [WATCH TIME: 5 minutes]

FDA panelists expressed concern over the drug's efficacy and data analysis, indicating the need for a confirmatory clinical trial.

The investigational antisense oligonucleotide, previously known as IONIS-C9Rx, failed to meet any of the secondary efficacy end points in its phase 1 trial but was well-tolerated. The companies plan to present the data at a future medical meeting.

The president and chief executive officer of The ALS Association provided insight on the availability of genetic testing for ALS and the organization’s efforts to increase access for the future. [WATCH TIME: 2 minutes]

Abnormal scores on cognitive testing persisted in 50% of patients without a pre-COVID history of cognitive abnormalities, irrespective of the presence or absence of a neurological complication during hospitalization.

The president and chief executive officer of The ALS Association highlighted a few of the major developments in the ALS field over the past decade, led by advances in technology. [WATCH TIME: 3 minutes]

Here's what is coming soon to NeurologyLive®.

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 headache and migraine.

Neurology News Network for the week ending March 26, 2022. [WATCH TIME: 4 minutes]

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