Neuromuscular Disorders
Conference Coverage
Steroids counter ataxia telangiectasia
Red blood cells infused with dexamethasone sodium phosphate delayed symptom progression and improved survival.
Conference Coverage
Nanoparticle shows promise for ALS
A phase 2 study revealed a significant survival advantage with drug that targets mitochondria.
FDA/CDC
FDA advisory panel rejects new ALS drug
The FDA panel raised a number of issues with the clinical trial, including the study’s small sample size and no survival benefit at 24 weeks.
From the Journals
Tremors and memory loss precede Parkinson’s in diverse population
Ethnically diverse study identifies prediagnostic Parkinson’s disease in primary care.
Latest News
Long COVID patients may develop nerve damage: Study
Ten patients – or 59% – had at least one test that confirmed neuropathy.
Latest News
Strep infection and tics in children: new data
If preteens present with a new-onset tic condition, “they’re unlikely to have it as a result of a group A streptococcal throat infection.”
Supplements
Newborn Screening for Spinal Muscular Atrophy in the United States
FDA/CDC
FDA approves new myasthenia gravis drug
This approval represents “an important step in providing a novel therapy option” for patients with the rare disease.
Latest News
Expected spike in acute flaccid myelitis did not occur in 2020
COVID-19 interventions may have reduced transmission of the associated enteroviruses.
Conference Coverage
A safer way to use Botox to treat challenging dystonia type?
A critical anatomical variation is still unrecognized by most clinicians performing botulinum toxin injections for oromandibular dystonia.
Conference Coverage
Investigative botulinum toxin formulation shows prolonged effect
A phase 3 trial reported that a new formulation of a botulinum toxin had a longer duration of effect with fewer side effects than existing...