Business Wire

Zatolmilast, an Investigational Treatment for Fragile X Syndrome, Receives Rare Pediatric Disease Designation from the U.S. FDA

OSAKA, Japan--(BUSINESS WIRE)--Shionogi & Co., Ltd. (Head Office: Osaka, Japan; Chief Executive Officer: Isao Teshirogi, Ph.D.; hereafter "Shionogi") announced the U ...
Local 12 WKRC Cincinnati

Twins with Fragile X syndrome able to live on their own in Point Arc home

CINCINNATI (WKRC) - A local program that allows people with disabilities to live independently has made a big difference in the Tri-State. It helped a set of twins living with Fragile X syndrome (FXS) ...
Business Insider

Anavex Life Sciences Announces Translational Biomarker Data for ANAVEX®2-73 (blarcamesine) in Fragile X Syndrome (Major Cause of Autism) at the 19th NFXF International Fragile ...

ANAVEX ® 2-73 corrects directly to humans’ translatable EEG biomarkers in a model of Fragile X Syndrome (FXS) Therapeutic potential to address behavioral and cognitive deficits in individuals with ...
Medical Xpress

Novel assay promises more efficient screening for carriers of fragile X syndrome

Researchers have developed an innovative new nanopore sequencing assay to identify carriers of fragile X syndrome (FXS), the leading cause of monogenic autism spectrum disorder and inherited ...
News Medical

Fragile X syndrome may be unfolding in brain cells even before birth

Fragile X syndrome, the most common form of inherited intellectual disability, may be unfolding in brain cells even before birth, despite typically going undiagnosed until age 3 or later. A new study ...
Business Wire

Harmony Biosciences Provides Update From Its Phase 3 RECONNECT Study of ZYN002 in Fragile X Syndrome

PLYMOUTH MEETING, Pa.--(BUSINESS WIRE)--Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY), today announced topline results from its Phase 3 registrational clinical trial (the RECONNECT Study) of ...
Morningstar

Mirum Pharmaceuticals Announces First Patient Enrolled in the BLOOM Phase 2 Clinical Study Evaluating MRM-3379 in Fragile X Syndrome

Proof-of-concept study underway for investigational oral therapy in Fragile X syndrome; top-line data from Phase 2 expected in 2027 Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease ...
Seeking Alpha

Harmony Biosciences Presents Promising Open-Label Extension Data of ZYN002 in Fragile X Syndrome

Data to be Presented at the Hot Topics in Child Neurology Podium Presentation Session at the American Academy of Neurology Annual Meeting PLYMOUTH MEETING, Pa.--(BUSINESS WIRE)-- Harmony Biosciences ...
MarketWatch

Spinogenix Announces Positive Type C Meeting with the FDA for SPG601 in Patients with Fragile X Syndrome (FXS)

The MarketWatch News Department was not involved in the creation of this content. -- Spinogenix has reached agreement with the FDA on important design elements for a Phase 2B/3 clinical trial of ...
abc27

European Medicines Agency Grants Orphan Drug Designation to Spinogenix's SPG601 for Treatment of Fragile X Syndrome (FXS)

LOS ANGELES, July 7, 2025 /PRNewswire/ -- Spinogenix, Inc., a clinical-stage biopharmaceutical company pioneering first-in-class therapeutics that restore synapses to improve the lives of patients ...
Nasdaq

Zynerba Pharmaceuticals Presents Long-Term Safety and Efficacy Data of Zygel™ in Children and Adolescents with Fragile X Syndrome (FXS) at the 2022 Society of Biological ...

DEVON, Pa., May 02, 2022 (GLOBE NEWSWIRE) -- Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare ...