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DeWine Announces Ohio First State in the Nation to Screen Newborn Babies for Duchenne Muscular Dystrophy
23 Apr 2024 22:00 GMT
… newborn babies for Duchenne Muscular Dystrophy (DMD).
The provision … cure; however, new treatments through gene therapy can … is well-positioned to treat children with DMD. … by the Parent Project Muscular Dystrophy advocacy organization: Nationwide Children …
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Duchenne UK and Parent Project Muscular Dystrophy Award $500,000 to Evaluate Safety and Tolerability of Muscle Progenitor Cells in Phase 1 Trial
23 Apr 2024 18:30 GMT
… dedicated to ending Duchenne muscular dystrophy (Duchenne), are … and won eight FDA approvals.
Everything we … drug development in their search to accelerate the development of treatments … -1-trial-302124699.html
SOURCE Parent Project Muscular Dystrophy (PPMD …
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First in the nation, Ohio to start screening newborns for Duchenne muscular dystrophy
23 Apr 2024 18:02 GMT
… country to screen for Duchenne muscular dystrophy (DMD) in newborns,” … no cure, but new treatments through gene therapy can … is well-positioned to treat children with this rare … by the Parent Project Muscular Dystrophy advocacy organization: Nationwide Children …
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First Clinical Trial Results of Gene Therapy (GNT0004) for Duchenne Muscular Dystrophy Presented at International Myology 2024 Congress
23 Apr 2024 17:00 GMT
… international multicenter gene therapy trial for Duchenne Muscular Dystrophy with GNT0004 product, … second dose.
Safety and pharmacodynamic results presented at Myology 2024 … combination with transient immunological prophylactic treatment, as well as efficacy …
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Edgewise Receives European Medicines Agency (EMA) Orphan Drug Designations for Sevasemten (EDG-5506) for the Treatment of Becker and Duchenne Muscular Dystrophies
23 Apr 2024 12:15 GMT
… Drug Designations for sevasemten for the treatment of Becker muscular dystrophy … drug designation to drugs and biologics intended for the treatment … Drug Administration (FDA) Orphan Drug Designation for the treatment … -finding clinical trial, assessing safety …
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Opinion: An FDA pathway can accelerate innovation for Duchenne muscular dystrophy
22 Apr 2024 11:48 GMT
… drug developers to continue improving it with innovation.
Charlie has Duchenne muscular dystrophy … with eteplirsen, the first treatment for Duchenne, which was … the Phase 3 clinical trial of a gene … controls.
Considering that cancer drugs that extend patients’ …
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First Patient Treated With Benitec Biopharma’s Oculopharyngeal Muscular Dystrophy Gene Therapy Shows Swallowing Improvements
21 Apr 2024 14:32 GMT
… therapy for the treatment of oculopharyngeal muscular dystrophy (OPMD)-related … results and continuing to treat patients as they … commonly prescribed GERD medications.
The investigational new drug (IND) … clinical trial was originally cleared by the FDA in …
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Catalyst Pharmaceuticals to Report First Quarter 2024 Financial Results on May 8, 2024
22 Apr 2024 12:00 GMT
… 2024 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. (“Catalyst” or “Company”) … with other medicines to treat primary generalized tonic-clonic … corticosteroid treatment for Duchenne Muscular Dystrophy. AGAMREE previously received FDA Orphan Drug and …
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'You can start treatments right away'; Arizona family pushes to get all newborns screened for deadly muscular dystrophy
19 Apr 2024 12:48 GMT
… Castle, 24, has Duchenne muscular dystrophy, a life-limiting muscle … said there are eight FDA approved treatments with more coming.
“ … And you can start these treatments right away. And it … In addition to starting treatment immediately, early detection would …
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Avance Clinical Showcases Clinical Excellence at World Orphan Drug Conference in Boston, April 23-25
20 Apr 2024 08:26 GMT
… Fragile X Syndrome, Duchenne Muscular Dystrophy, Rett Syndrome and Erythropoietic … experience in delivering FDA and EMA-quality data … partner to accelerate their drug development programs,” Lungershausen said … s team of clinical trial experts, including Senior …