Author Archives: Forest Ray PhD

Ionis Pharmaceuticals intends to launch a clinical trial later this year to test ION582, a potential Angelman syndrome therapy.  The Phase 1/2a trial, designed to enroll up to 50 patients, is expected to start in the second half of 2021, Ionis wrote in a letter to the…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

Children with Angelman syndrome may be more prone to developing cavities in their teeth, due to the growth of thin enamel with an abnormal protein content, according to a new study. The study, “The effect of Angelman syndrome on enamel and dentin mineralization,” was published in…

Healx and the Foundation for Angelman Syndrome Therapeutics (FAST) are partnering to identify new therapies for Angelman syndrome from among already-approved and available medications, according to a company press release. Finding new uses for old medications — a process called drug repositioning or repurposing —…

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in the United States. People with rare diseases know that the impacts of such conditions extend beyond just medical…

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will support activities that align with the foundation’s Rare Belonging focus, a set of funding priorities aimed at improving the…

The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for a disease that few people — sometimes even physicians — have heard of is challenging on its own merit.

GTX-102, a potential treatment for Angelman syndrome, is cleared for clinical trials, thanks to the activation of its investigational new drug (IND) application by the U.S. Food and Drug Administration. Enrollment in an upcoming Phase 1/2 trial is due to begin in the first half of…