Rare Diseases
Article
NORD Publishes Three New Free Rare Disease Guides for Physicians
Conference Coverage
Fenfluramine trials in Dravet syndrome yield highly positive results
WASHINGTON – Adjunctive fenfluramine reduced mean monthly convulsive seizure frequency by 63.9% vs. placebo and had no evidence of cardiotoxicity...
Article
Morgan Leary Vaughan Fund Receives Petit Family Foundation Grant
Article
Bridge the Gap – SYNGAP Education and Research Foundation
Article
Small Kansas Town Turns Night Into Day for Boy With Rare Disease
Article
NORD Supports Orphan Drug Tax Credit
Article
NORD Board Chair to Moderate Social Security Administration Forum
Article
NORD Call for Abstracts Reopened
Article
Orphan Drugs Are Not Driving Up the Cost of Health Care, According to New Study
Article
Nominate a Colleague or Patient for a NORD Rare Impact Award
From the Journals
Gene therapy for cerebral adrenoleukodystrophy shows promise
A majority of boys had stabilization of lesion progression and no major functional disability.