Rare Diseases
Article
FDA Commissioner Comments on Signing of the Right to Try Act
SILVER SPRING, MD—On May 30, 2018, President Donald Trump signed into law the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew...
Conference Coverage
Myelin antibody predicts ADEM relapse
LOS ANGELES – Could results help to guide decisions on immunosuppressive treatment?
Conference Coverage
Guidance coming for mTOR inhibitors in infantile TSC
LOS ANGELES – Survey aimed to describe the wide variation in their off-label use, particularly in infants.
Audio
Mohammed Sanjak, PhD
Dr. Sanjak describes a proposed test for assessing lower-limb muscle strength in patients with ALS.