Rare Disease Report

Top 5 Rare Disease News of the Week—June 17, 2018

JUNE 23, 2018
Rare Disease Report® Editorial Staff

#5: Researchers Prove Rasmussen’s Encephalitis Is an Autoimmune Disease

New evidence suggests that Rasmussen’s encephalitis is an autoimmune disease.

The degenerative disease most often develops in pediatric patients with unknown causes and presents with recurring epileptic seizures that resist traditional treatments. In serious cases, the frequency of seizures is reduced by separating the two brain hemispheres—or removing one hemisphere altogether—and coping with the subsequent neurological consequences.

Read more about Rasmussen’s encephalitis.

#4: FDA Grants Orphan Drug Designation to New Treatment for Rett Syndrome

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to AMO Pharma Limited’s investigational therapy AMO-04 for the treatment of Rett syndrome, a rare childhood neurodevelopmental disorder.

AMO-04 is a glutamate modulator; it has displayed early-stage promise for Rett syndrome treatment based on screening by the Scout Program, which is a drug discovery screen in a mouse model of the disease sponsored by Rettsyndrome.org.

Read more about the new treatment for Rett syndrome.

Stay informed on the latest rare disease news and developments by signing up for our newsletter.
Copyright © RareDR 2013-2019 Rare Disease Communications. All Rights Reserved.