Rare Diseases
News from NORD
Research Training Fellowship in Ataxia is Available
Literature Review
Can Coenzyme Q10 Supplementation Benefit Patients With MSA?
Patients with multiple system atrophy (MSA) have decreased levels of plasma coenzyme Q10 (CoQ10), regardless of their COQ2 genotype,...
Commentary
Myth of the Month: Vaccinations in patients with Guillain-Barré syndrome
Is there good evidence for patients with a history of Guillain-Barré syndrome to avoid influenza vaccines or vaccinations in general?
News from NORD
NORD Rare Diseases and Orphan Products Summit to Feature Speakers from FDA, NIH, and ACMG
News from NORD
NORD Issues Statement as US Senate Postpones Vote on Cures Legislation
News
Hemophilia drugs top Medicaid spending per prescription
Medicaid’s three most expensive drugs by spending per prescription were for treatment of hemophilia, with the cost leader coming in at almost $59,...
News
Intermediate alleles may confer mild, late-onset Huntington-like symptoms
Key clinical point: Asymptomatic people who carry an intermediate number (27-35) of CAG repeats on the huntingtin gene may be at increased risk...
Conference Coverage
Updated Behçet’s disease recommendations expand biologic treatment
Conference Coverage
Heat shock protein peptide vaccine appears safe, effective for glioblastoma patients
Key clinical point: A heat shock protein peptide vaccine appears safe and effective for patients with glioblastoma in an early stage trial. Major...
Conference Coverage
Edaravone slows progression of ALS when started early on
Key clinical point: When added to standard of care, edaravone slows loss of function in patients with early-stage ALS. Major finding: Compared...
Conference Coverage
Temozolomide + RT boosts survival in elderly with glioblastoma
Key clinical point: Combining temozolomide with radiotherapy prolongs survival for elderly with glioblastoma. Major finding: Combining...