Rare Diseases
News from NORD
More Than 100 Patient Organizations Join NORD in Supporting the RARE Act
News from NORD
RFPs Available for the Study of Three Rare Diseases
News from NORD
2018 Marks 35th Anniversary of NORD and the Orphan Drug Act
News from NORD
Register Now for NORD’s Rare Impact Awards Celebration
Conference Coverage
Intraventricular enzyme replacement slows CLN2 disease progression
LOS ANGELES – “Durable and persistent” benefit seen in open-label trial of cerliponase alfa in neuronal ceroid lipofuscinosis type 2 disease.
Daily News Podcast
MDedge Daily News: Which nonopioids are ripe for abuse?
And bum kidneys shouldn’t stop dabigatran reversal.
Conference Coverage
VIDEOS: High-priced drugs, out-of-pocket costs raise challenges for neurologists
LOS ANGELES – Members of the AAN Neurology Drug Pricing Task Force spoke about the issues that arise in getting patients access to high-cost drugs...
Feature
‘Right to try’ bill passes House
The bill passed despite opposition from a broad coalition of physician and patient groups.
Feature
FDA approves subcutaneous immunoglobulin treatment for CIDP
Hizentra is the first FDA-approved subcutaneously administered human immunoglobulin maintenance therapy for CIDP.
Conference Coverage
No Consensus Treatment for Neurosarcoidosis
NAPLES, FL—Although patients with symptomatic neurosarcoidosis account for approximately 5% to 15% of people with systemic sarcoidosis, making it...
Conference Coverage
New Drug Technology Could Treat Huntington’s Disease
SAN DIEGO—Developments in strategies based on CRISPR and antisense oligonucleotides (ASOs) have raised the prospect that these therapies targeting...