Rare Diseases
Conference Coverage
OV-101 shows promise for Angelman syndrome
A novel drug affecting the GABAergic system improved sleep problems in a sample of adults and adolescents with Angelman Syndrome.
Literature Review
Cognitive and Behavioral Problems Increase With ALS Disease Stage
Cognitive deficits and behavioral symptoms that are specific to amyotrophic lateral sclerosis (ALS) occur more...
FDA/CDC
FDA approves Sympazan for Lennox-Gastaut syndrome
Patients with Lennox-Gastaut syndrome can have difficulty swallowing pills or large-volume oral suspensions, so this oral film may reduce care...
Conference Coverage
Triggers May Guide Treatment of New Daily Persistent Headache
ASHEVILLE, NC—New daily persistent headache is rare and difficult to treat. Although neurologists may be tempted to try...
Conference Coverage
What Are the Clinical, Laboratory, and Electrodiagnostic Features of Zinc Deficiency-Induced Peripheral Neuropathy?
WASHINGTON, DC—Patients with zinc deficiency-induced peripheral neuropathy may present with paresthesia, gait...
Conference Coverage
Does Thymectomy Benefit Patients With Anti-MuSK Myasthenia Gravis?
WASHINGTON, DC—Among patients with anti-muscle-specific kinase (MuSK) myasthenia gravis, thymectomy is not associated...
Conference Coverage
Disability in Patients With Stiff Person Syndrome May Progress Faster Than Thought
WASHINGTON, DC—Stiff person syndrome leads to disability if therapy is not initiated early in the disease course,...
News from the FDA/CDC
FDA approves Xyrem to treat children with narcolepsy
The central nervous system depressant was approved for the treatment of cataplexy and excessive daytime sleepiness.
Video
Technology offers new tools, challenges for rare-disease patients
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Video
Education and support enhance care for rare-disease patients
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Conference Coverage
New Registry Offers Insight Into Opsoclonus-Myoclonus Syndrome
WASHINGTON – Most patients with opsoclonus myoclonus syndrome experience ataxia, among multiple other symptoms.