Neuromuscular Disorders
Literature Review
First-ever Huntington staging system may jump-start drug development for early-stage disease
Researchers liken the Huntington’s disease Integrated Staging System (HD-ISS) to the system currently used to stage cancer.
FDA/CDC
FDA unveils 5-year plan for ALS and other neurodegenerative diseases
“We recognize the urgent need for new treatments that can both improve and extend the lives of people diagnosed with these diseases.”
Conference Coverage
Tofersen linked to slow, positive effects in ALS
One-year clinical trial results show a deceleration in functional decline that is similar, but “more pronounced” than the previously reported 6-...
FDA/CDC
FDA expands indication for spinal muscular atrophy drug
After 12 months of risdiplam treatment, the majority of presymptomatic infants with SMA reached key motor milestones.
FDA/CDC
FDA approves oral form of ALS drug edaravone
Compared with the IV formation of the drug, the oral form has been shown to generate comparable levels of active drug in the bloodstream.
Recap
Reducing Attacks and Neuropathic Pain in NMOSD
Relapse and severity of attack-associated neuropathic pain are strong predictors of quality of life in patients with NMOSD. Dr Michael...
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.