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MIRM Enrolls First Patient in Phase II Fragile X Syndrome Study
Mirum Pharmaceuticals MIRM announced that it has enrolled the first patient in the phase II BLOOM study evaluating its newly ...
Nearly a year and a half ago, I penned my final “From the Editor” column for Lowcountry Parent, sharing publicly for the first time about my son Archer’s diagnosis of Fragile X syndrome at 3 years old ...
Fragile X Syndrome: Symptoms, Diagnosis, Treatments and Complications: By Shreoshree Chakrabarty Fragile X Syndrome (FXS), also known as FX syndrome or fragile syndrome, is the most common inherited ...
OSAKA, Japan--(BUSINESS WIRE)--Shionogi & Co., Ltd. (Head Office: Osaka, Japan; Chief Executive Officer: Isao Teshirogi, Ph.D.; hereafter "Shionogi") announced the U ...
For 22 years, Jason Mazzola's life was defined by Fragile X, a genetic condition that often causes autism and intellectual disability. Jason, who is 24 now, needed constant supervision. He had ...
LOS ANGELES, Jan. 13, 2025 /PRNewswire/ -- Spinogenix, Inc., a clinical-stage biopharmaceutical company pioneering first-in-class therapeutics that restore synapses to improve the lives of patients ...
(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 ...
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Southbury mom opens Dripability apparel, giving work experience to young adults with special needs
It’s a chance for them to experience a taste of the working world” and enjoy "meaningful" employment, Marla Wayne said.
They called him “the singing baby.” As a newborn, Maxwell Wheeler would lie in his crib, whistling shrilly as he breathed in and out. For Cari and Andrew Wheeler of Madera Ranchos, Calif., it was one ...
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