Mutations in FMR1, the gene encoding fragile X messenger ribonucleoprotein 1 (FMRP), located at Xq27.3 and discovered in 1991, are the leading single-gene causes of autism and intellectual disability.
ESCONDIDO, Calif.--(BUSINESS WIRE)--Enthorin Therapeutics, LLC, a biotechnology company focused on circuit-modulating treatments for neurological and neurodevelopmental disorders, announced that its ...
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Fragile X syndrome: Study details cellular and molecular activity that leads to cognitive chaos
The genetic disorder Fragile X syndrome occurs when individuals don't make the Fragile X protein known as FMRP. Essential for normal brain development, FMRP helps control when and where proteins are ...
Servier is souping up its neurology pipeline with a clinical-stage candidate sourced from U.K. biotech Kaerus Bioscience for a rare genetic disease. The French pharma is offering as much as $450 ...
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 ...
PLYMOUTH MEETING, PA — Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) reported topline results from its Phase 3 RECONNECT study of ZYN002 in Fragile X syndrome (FXS), announcing that the trial did ...
Enthorin Therapeutics Announces Initiation of Phase II Clinical Trial of MRM-3379 (Formerly ENT-3379) for Fragile X Syndrome by Licensing Partner Mirum Pharmaceuticals Enthorin Therapeutics, LLC, a ...
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