Fragile X News Today

January 14, 2022January 14, 2022

Nova Mentis, Mycrodose Team Up to Test Skin Patch Delivery of Psilocybin

Nova Mentis and Mycrodose Therapeutics have teamed up to test whether psychedelic treatments, such as Nova’s psilocybin — a potential behavioral therapy for fragile X syndrome — can be delivered ... Read more

January 7, 2022January 7, 2022

Zygel Cannabidiol Gel Tolerated Well Up to 21 Months, Trial Data Show

Fragile X News Today

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 ... Read more

December 17, 2021December 17, 2021

$100K Donor-matching Grant Announced to Find Fragile X Treatments

Fragile X News Today

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 ... Read more

December 10, 2021December 10, 2021

Wearable Motion Sensors May Help Diagnose Fragile X

Fragile X News Today

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 ... Read more

December 3, 2021December 4, 2021

Study to Look at Grammar, Executive Functioning in Children With Fragile X

Fragile X News Today

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 ... Read more

November 19, 2021November 19, 2021

Quris Planning AI-powered Clinical Trial in 2022

Fragile X News Today

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 ... Read more

November 12, 2021November 12, 2021

Rare Disease Groups in US Join in Plea for Care Across State Lines

COVID-19 News, Fragile X News Today

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 ... Read more

November 5, 2021November 5, 2021

Nova Seeks Orphan Drug Status in US for Psilocybin, Hallucinogenic Therapy

Fragile X News Today

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 ... Read more

October 29, 2021October 29, 2021

FDA Agrees to Phase 2 Trial of HLX-0201 in Treating Fragile X

Fragile X News Today

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, ... Read more

October 22, 2021October 22, 2021

UC Davis Institute Offers COVID-19 Vaccines in Clinic Tailored for Fragile X

COVID-19 News, Fragile X News Today

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 ... Read more

October 21, 2021October 21, 2021

Global Genes, Diversity Coalition Team Up to Advance Health Equity

Fragile X News Today

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, ... Read more

October 15, 2021October 15, 2021

Patients Ages 3–17 Sought for Large-Scale Trial Testing CBD Gel Zygel

Fragile X News Today

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 ... Read more

October 14, 2021October 14, 2021

Rare Disease Diversity Coalition Awards $600K to Combat Disparities

COVID-19 News, Fragile X News Today

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 ... Read more

October 8, 2021October 8, 2021

‘Rare’ Documentary in Kickstarter Campaign to Raise $45K by Oct. 28

Fragile X News Today

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 ... Read more

October 1, 2021October 1, 2021

CTH120 Medication Granted Orphan Designation in EU

Fragile X News Today

Connecta Therapeutics‘ CTH120 molecule has been granted orphan designation by the European Medicines Agency (EMA) for treating fragile X syndrome, the leading cause of hereditary intellectual disability and autism spectrum ... Read more