Rare Diseases
Conference Coverage
Inebilizumab reduces the risk of NMOSD attacks
Philadelphia – The humanized monoclonal antibody was well tolerated and reduced the risk of disability worsening.
Conference Coverage
Can an antisense oligonucleotide benefit patients with SOD1-ALS?
PHILADELPHIA – In patients with amyotrophic lateral sclerosis caused by mutations in the SOD1 gene, an investigational treatment reduced SOD1...
FDA/CDC
Ruzurgi approved for Lambert-Eaton myasthenic syndrome in patients under age 17
The approval is the first for a LEMS treatment specifically for pediatric patients.
Conference Coverage
Angelman syndrome treatment safe, well-tolerated, and effective in exploratory analyses
PHILADELPHIA – Experimental treatment may benefit adolescents and adults with Angelman syndrome.
Latest News
CSF and plasma biomarkers predict survival in sporadic Creutzfeldt-Jakob disease
An analysis suggests the possibility of using a minimally invasive biomarker to guide clinical care and improve clinical trial design.
Conference Coverage
Neurodevelopmental concerns may emerge later in Zika-exposed infants
BALTIMORE – Some Zika-exposed children showed poorer mobility and social cognitive scores as they approached 18 months even though their fetal...
Conference Coverage
Is pro soccer a risk factor for ALS?
A higher risk of ALS and an earlier age of onset was found among professional soccer players.
Literature Review
Polyglutamine diseases are rare, but not the mutations that cause them
A population-based study found more than 10% of European adults carried CAG repeats in the pathogenic range, despite lacking signs of disease.
Latest News
Trofinetide may benefit patients with Rett syndrome
Data from a phase 2 study expand on previous findings and suggest that trofinetide can alleviate core symptoms of the neurodevelopmental disorder...
Medical Education Library
Rare Neurological Disease Special Report
Conference Coverage
Dr. Lisa Christopher-Stine: Polymyositis? It’s more likely something else
Thinking polymyositis? Look closely for IMNM, overlap with other rheumatologic conditions, antisynthetase syndrome, inclusion body myositis, and...