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Rare Diseases
Out-of-Pocket Costs Steadily Increasing for Neurologic Care
January 05, 2021
Article
A recent analysis of UnitedHealthcare insurance claims revealed increases of almost 200% for some services between 2001 and 2016.
MOVE-FA Study Initiated for Friedriech’s Ataxia in Children and Young Adults
December 23, 2020
Article
PTC Therapeutics announced the initiation of its third study of 2020 investigating vatiquinone.
Initial Results Prove Positive for Low-Dose AXO-AAV-GM1 in GM1 Gangliosidosis
December 16, 2020
Article
These data follow a recent announcement from Sio Gene Therapies that the first patient in the high-dose cohort has been dosed.
Mary Schroth, MD: Cure SMA’s Ongoing Advocacy Initiatives
September 15, 2020
Video
The chief medical officer at Cure SMA spoke to the ongoing work that the organization does in advocating for patients with spinal muscular atrophy, particularly for newborn screening.
GW Submits sNDA for CBD in Tuberous Sclerosis Complex
February 07, 2020
Article
This additional application, supported by data from the GWPCARE6 trial, seeks to expand the indication of cannabidiol to include seizures associated with tuberous sclerosis complex.
Satralizumab BLA Accepted by FDA for Treatment of NMOSD
November 13, 2019
Article
Study data demonstrates that treatment with satralizumab reduces risk of relapses in patients with neuromyelitis optica spectrum disorder.
FDA Rejects Duchenne Muscular Dystrophy Drug Golodirsen, Citing Safety Concerns
August 23, 2019
Article
The regulatory agency cited 2 concerns in the complete response letter: the risk of infections related to intravenous infusion ports and renal toxicity.
Developing Targeted Therapeutics in Angelman Syndrome
July 16, 2019
Article
The genomic revolution has led to increasing opportunities to address complex neurodevelopmental conditions, taking a genetics-first approach.
Genetic Testing for Huntington Disease
July 03, 2019
Article
The profound consequences of the results guide decisions about testing. Who should be tested? And, just as important, who should not?
Phase 2 Trial for Angelman Syndrome Underway
June 25, 2019
Podcast
In this podcast, Heidi Moawad, MD interviews Cesar Ochoa-Lubinoff, MD, MPH, FAAP, about recent encouraging clinical trial results in patients with Angelman syndrome.