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Zygel, an experimental cannabidiol gel that Zynerba Pharmaceuticals is developing to help normalize behaviors in people with fragile X syndrome, continues to be well tolerated up to a median treatment length of 21 months, according to new data from the open-label extension of the Phase 2/3 CONNECT-FX trial.
To help find new fragile X treatments, the Robert & Ardis James Foundation has presented the Fraxa Research Foundation with a donor-matching challenge of up to $100,000. All funds raised will go toward research to develop therapies, and ultimately a cure, for fragile X, a genetic condition associated…
Wearable motion sensors someday may help doctors diagnose fragile X syndrome and similar disorders based on the way people walk, a study suggests. The method may be able to identify problems in walking 15 to 20 years ahead of clinical symptoms; as such, it offers an opportunity to intervene sooner to…
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…
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