Discover Fragile X Syndrome (FXS)- the causes, symptoms, diagnosis, and treatment options. Learn about FXS signs, care tips, ...

Harmony also touts late-stage asset EPX-100 (clemizole hydrochloride), which the biotech is running through a pair of phase 3 trials in Dravet syndrome and Lennox-Gastaut Syndrome— both rare forms of ...

Building on more than two decades of research, a study by MIT neuroscientists at The Picower Institute for Learning and Memory reports a new way to treat pathology and symptoms of fragile X syndrome, ...

Research on fragile X syndrome, the most common inherited cause of mental retardation, has focused mostly on how the genetic defect alters the functioning of neurons in the brain. A new study focusing ...

Harmony Biosciences has paused a mid-stage trial of ZYN002 in 22q11.2 deletion syndrome after the THC-free cannabinoid drug ...

Mirum Pharmaceuticals (($MIRM)) announced an update on their ongoing clinical study. Study Overview: Mirum Pharmaceuticals is conducting a Phase 2 ...

SPG601, Granted Fast Track Designation by the FDA, Reduced Signature Changes in High-Frequency Gamma Band Activity in Fragile X Syndrome Patients, a Common Inherited Form of Intellectual Disability ...

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 ...

Please provide your email address to receive an email when new articles are posted on . The FDA has granted fast-track designation to an investigational small molecule to treat individuals with ...

(RTTNews) - Harmony Biosciences Holdings, Inc. (HRMY), Tuesday announced the updated data from its Open-Label Extension or OLE study ZYN2-CL-017, evaluating the safety and effectiveness of ZYN002 in ...

LOS ANGELES, Oct. 22, 2025 /PRNewswire/ -- Spinogenix, Inc., a clinical-stage biopharmaceutical company pioneering first-in-class therapeutics that restore synapses to improve the lives of patients ...