Neuromuscular Disorders
FDA/CDC
FDA okays drug for Duchenne muscular dystrophy
The approval of vamorolone “provides people living with Duchenne, and their families, a powerful tool to treat the disease, while limiting some...
Rare Diseases Report 2023
Stiff person syndrome: When a rare disorder hits the headlines
Awareness of this disorder is increasing, but clinicians are challenged to apply the proper workupto avoid wrong turns in identifying affected...
Rare Diseases Report 2023
The dawning age of therapy for Friedreich ataxia
The first therapy to target the underlying pathology of Friedreich ataxia was approved in 2023. Other drug and genetic therapies are in the...
Rare Diseases Report 2023
Has prompt diagnosis of amyotrophic lateral sclerosis become urgent?
Optimism is high about improving the survival and care of ALS patients. Neurologists who don’t specialize in ALS can add to the positivity by...
Rare Diseases Report 2023
Emerging therapies in Duchenne and facioscapulohumeral muscular dystrophy
Newly approved and investigational therapies, and enhanced diagnostics, are sparking optimism about treating MD especially Duchenne and...
Feature
Are cellular therapies the future of autoimmune disease?
Promising results from small case studies and early clinical trials hint at the potential of CAR T-cell therapy to treat – and possibly cure –...
FDA/CDC
Myasthenia gravis drug gets FDA nod
Rozanolixizumab is the first FDA-approved treatment for adults with both subtypes.
Conference Coverage
U.S. incidence, prevalence of myasthenia gravis is rising
These new estimates are “significantly higher” than those from a prior U.S. analysis from 2003.
Conference Coverage
Therapy to reverse muscle dystrophies shows promise
Interim study data showed rapid reductions in multiple biomarkers of muscle injury, reductions in anti-inflammatory markers, proteomic changes...
From the Journals
Childhood trauma tied to increased Parkinson’s disease severity
The relationship between childhood trauma and PD appears to be dose dependent, new research shows.
Feature
Physician pleads guilty to 52 counts in opioid scheme
The neuromuscular medicine specialist also admitted to ignoring patient requests to lower dosages and signs that patients were selling prescribed...