Betty Vertin is a mother, writer and a fierce defender of the decisions she and her husband Jason made about the seven children they brought into this world. Betty writes…
As reported on PharmaBiz, Regenxbio has announced encouraging topline findings from the pivotal Phase III portion of its ongoing AFFINITY DUCHENNE clinical program evaluating RGX-202, an investigational gene therapy for…
As reported on BioPharmaDive, Roche is restarting efforts to get Elevidys, a gene therapy for Duchenne muscular dystrophy (DMD), approved in Europe by launching a new global Phase 3 trial…
As reported on the Manila Times, Aucta Pharmaceuticals has officially introduced PYQUVI™ (deflazacort) oral suspension 22.75 mg/mL, marking the company’s entry into the U.S. commercial branded‑generic market. The therapy, released…
Brussels — On February 24, 2026, the acclaimed Netflix documentary The Remarkable Life of Ibelin received the Media & Awareness Raising Award at the EURORDIS Black Pearl Awards. The honor…
NS Pharma, Inc., a subsidiary of Nippon Shinyaku Co., Ltd., has received a significant regulatory boost for its experimental treatment targeting Duchenne muscular dystrophy (DMD). As reported in The AI…
ITF Therapeutics has announced the publication of encouraging long-term data supporting the efficacy and safety of givinostat as a treatment for Duchenne muscular dystrophy (DMD), according to a recent article…
CureDuchenne has launched the CureDuchenne Biobank, a neutral, centralized and self-funded data hub that will unlock information to accelerate research. It will provide a consistent set of resources for scientific…
According to a report by biopharmadive.com, Pfizer announced they will be dropping support for an experimental Duchenne muscular dystrophy treatment. The company intends to see if the treatment may be…
What can one little boy and his family do to fight a rapidly debilitating and (currently) incurable disease such as Duchenne muscular dystrophy? A lot. The diagnosis of a loved…
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