News

A five-year, $2.4 million grant will fund a new study of grammar skills and executive functions in preschool and school-age children with fragile X syndrome. The grant, awarded by National Institute on Deafness and Other Communication Disorders, part of the National Institutes of Health, will be…

A candidate therapy for fragile X syndrome — developed using Quris’ bio-artificial intelligence clinical prediction platform — will enter clinical trials in 2022, the company announced. Quris is using the power of artificial intelligence (AI)  combined with a “patients-on-a-chip” platform to predict the safety and potential effectiveness of therapeutic…

Over 230 national organizations signed a letter urging all 50 U.S. state governors to “maintain and expand” flexibility with licensure requirements for the duration of the COVID-19 pandemic to ease access to care. During the pandemic, governors used emergency authority to waive certain state licensure requirements, giving healthcare providers…

Nova Mentis has filed an application for orphan drug designation with the U.S. Food and Drug Administration (FDA) for psilocybin, its investigational psychedelic therapy for fragile X syndrome. Recently, psilocybin received orphan drug designation from the European Medicines Agency (EMA), which offers financial incentives for potential rare disease treatments. Nova also…

A Phase 2a clinical trial of HLX-0201 in treating behavioral symptoms in people with fragile X syndrome was given U.S. Food and Drug Administration (FDA) approval to open, the therapy’s developer, Healx, announced. Recruitment for the trial (NCT04823052) should begin in the coming months, the company stated in a…

Many COVID-19 clinics are large, crowded, and on the boisterous side, which can be overwhelming for people with fragile X syndrome and other neurodevelopmental disorders needing to go there for vaccination. Such concerns led the UC Davis MIND Institute in Sacramento, California, to open a custom-designed clinic for vaccine administration. The…

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…

Zynerba Pharmaceuticals is recruiting children and adolescents with fragile X syndrome for a large-scale clinical trial testing the safety and efficacy of the cannabidiol (CBD) gel Zygel for treating behavioral problems. The Phase 3 trial, called RECONNECT, is enrolling participants ages 3—17 at 26 sites across the U.S., Australia,…

The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC steering committee working groups, which aim to identify problems for rare disease communities and advocate for solutions. The five…

A crowdfunding campaign aims to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and Digital Cave Media launched the campaign — allowing filmmakers to finish producing and to release the documentary — on Kickstarter.