Neuromuscular Disorders
Rare Diseases Report 2022
Novel gene-based therapies for neuromuscular diseases
Novel gene-based therapies show significant potential for transforming the treatment of neuromuscular diseases.
Rare Diseases Report 2022
Spinal muscular atrophy: Patient care in the age of genetically targeted therapy
Newborn screening, updated treatment guidelines, and treatment algorithms have changed what can be clinically done for patients with SMA, but...
From the Journals
Racial disparities in preventive services use seen among patients with spina bifida or cerebral palsy
Comorbidities were highest among Black patients in a new study.
FDA/CDC
ALS drug gets FDA panel thumbs-up after rare second look
FDA officials in the meeting stressed the importance of considering unmet medical need in ALS in the panel’s decision-making process.
Hitting a Nerve
Polio: The unwanted sequel
Cracks in herd immunity have allowed polio to return.
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...