Multicenter expanded access program for access to investigational products for amyotrophic lateral sclerosis.

Introduction/aims: Expanded access (EA) is a Food and Drug Administration-regulated pathway to provide access to investigational products (IPs) to individuals with serious diseases who are ineligible for clinical trials. The aim of this report is to share the design and operations of a multicenter, multidrug EA program for amyotrophic lateral sclerosis (ALS) across nine US centers.

Methods: A central coordination center was established to design and conduct the program.

Dextromethorphan/quinidine for the treatment of bulbar impairment in amyotrophic lateral sclerosis.

Objective: No efficacious treatments exist to improve or prolong bulbar functions of speech and swallowing in persons with amyotrophic lateral sclerosis (pALS). This study evaluated the short-term impact of dextromethorphan/quinidine (DMQ) treatment on speech and swallowing function in pALS.

Methods: This was a cohort trial conducted between August 2019 to August 2021 in pALS with a confirmed diagnosis of probable-definite ALS (El-Escorial Criteria-revisited) and bulbar impairment (ALS Functional Rating Scale score ≤ 10 and speaking rate ≤ 140 words per minute) who were DMQ naïve.

Randomized trial of inosine for urate elevation in amyotrophic lateral sclerosis.

Introduction/aims: Higher urate levels are associated with improved ALS survival in retrospective studies, however whether raising urate levels confers a survival advantage is unknown. In the Safety of Urate Elevation in Amyotrophic Lateral Sclerosis (SURE-ALS) trial, inosine raised serum urate and was safe and well-tolerated. The SURE-ALS2 trial was designed to assess longer term safety.

Provisional best practices guidelines for the evaluation of bulbar dysfunction in amyotrophic lateral sclerosis.

Introduction: Universally established comprehensive clinical bulbar scales objectively assessing disease progression in amyotrophic lateral sclerosis (ALS) are currently lacking. The goal of this working group project is to design a best practice set of provisional bulbar ALS guidelines, available for immediate implementation within all ALS clinics.

Methods: ALS specialists across multiple related disciplines participated in a series of clinical bulbar symposia, intending to identify and summarize the currently accepted best practices for the assessment and management of bulbar dysfunction in ALS Results: Summary group recommendations for individual speech, Augmentative and Alternative Communication (AAC), and swallowing sections were achieved, focusing on the optimal proposed level of care within each domain.

Primary Diffuse Leptomeningeal Melanomatosis: Case Report and Review of the Literature.

Background: Primary diffuse leptomeningeal melanomatosis (PDLM) is an extremely rare pathologic condition that can mimic several other neurologic disease states.

Methods: We report a rare case of PDLM without evidence of a primary focus. In addition, we performed a comprehensive review of the literature to describe all previously reported cases of PDLM.

Best practices protocol for the evaluation of bulbar dysfunction: summary recommendations from the NEALS bulbar subcommittee symposium.

Objective: The aim of this Symposium was to develop a consensus based, bulbar assessment protocol for implementation within NEALS clinics.

Methods: A one-day symposium, held in April 2017, was organized into Speech and Swallowing sections to establish summary recommendations for the assessment of bulbar dysfunction within each group.

Results: Summary recommendations included speech referrals and AAC evaluations at initial visit, CNS-BFS, maximum sustained phonation, and speaking rate.

Ictal asystole: a benign condition?

Ictal asystole (IA) has been implicated as a preventable cause of sudden unexplained death in epilepsy presumably provoked by a direct autonomic effect of the electrical stimulus on the heart. An electronic database search of patients with IA was performed comparing heart rate (HR) characteristics to a group of patients with vasovagal asystole. IA was seen in eight patients, all with temporal lobe epilepsy.

Myasthenia gravis improvement after laparoscopic Roux-en-Y gastric bypass.

Many diseases in the obese population have been found to improve after weight loss. A 56-year-old female with a long history of myasthenia gravis (MG) and morbid obesity is reported. Preoperatively, she presented with a BMI of 46.