Mary Chapman, features writer —

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The initial phase of the five-year, $1-million #RAREis Scholarship Fund will include 32 scholarships — each totaling $5,000 —…

In light of the COVID-19 pandemic, there are several ways to get involved virtually to support Dravet Syndrome Awareness Month, observed each June. Organized by the Dravet Syndrome Foundation (DSF), the monthlong event is intended to make this severe type of epilepsy — and the needs of…

The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. Survey questions cover a patient’s physical and mental health, supply of treatments, and access to healthcare, among other…

The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. Called the NORD COVID-19 Critical Relief Program, the effort will provide up to $1,000 annually to those eligible to…

In a move that will give Dravet syndrome patients easier treatment access, the U.S. Drug Enforcement Administration (DEA) has reclassified Epidiolex (formerly GWP42003-P) as a non-controlled substance. In what the agency called a “descheduling,” the action removes all federally controlled-substance restrictions for the oral cannabidiol (CBD) solution that…

Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, and aims to supports work by researchers and clinicians into a better understanding of and treatments for rare diseases, as well…

An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…

To speed diagnoses and provide free genetic testing for young children in the United States and Canada who have unexplained seizures, BioMarin Pharmaceutical and Invitae have expanded their Behind the Seizure program. New partners in the effort include Biogen, Encoded Therapeutics, Neurogene, Praxis…

In a departure from general government policy, Japan has approved the cannabis compound Epidiolex for clinical trials in epileptic patients with Dravet and Lennox-Gastaut syndromes. Although the country prohibits cannabis-based agents overall, tightly monitored hospital trials are exempt from its Cannabis Control Act. Epidiolex is a plant-derived…

The National Institute of Neurological Disorders and Stroke (NINDS) has awarded NeuroCycle Therapeutics a $500,000 grant to test advanced subtype-selective GBAA receptor modulators in Dravet syndrome models. The Small Business Innovation Research grant to evaluate NCT10004 and NCT10015 furthers the pharmaceutical company’s efforts…