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Agamree for Duchenne muscular dystrophy available by prescription in US
18 Mar 2024 21:51 GMT
… treat Duchenne muscular dystrophy in those 2 years and up. Image: Adobe Stock
FDA … muscular dystrophy treatment, offering hope and improved quality of life for patients,” Catalyst Pharmaceuticals …
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Michael Kelly, PhD, on Gene Therapy’s Continuing Path to Treating DMD
18 Mar 2024 14:49 GMT
… more about how the treatment field for DMD has … Therapeutics announces U.S. FDA acceptance of an efficacy … ;sarepta-therapeutics-announces-us-fda-acceptance-efficacy?_ga=2 … gene therapy for Duchenne muscular dystrophy. News release. Sarepta Therapeutics …
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Loss of man during clinical trial propels work at new rare diseases lab in Woodbridge
18 Mar 2024 12:56 GMT
… Terry’s devastating disease.
“Duchenne muscular dystrophy is a fatal progressive neurological … a clinical trial was given the green light by the FDA.
In … October of 2022, Terry received the treatment and …
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Aurobindo Pharma arm announces successful Phase 1 clinical study outcome of their BP11 product
18 Mar 2024 08:24 GMT
…
The PK/PD trial was conducted in 165 healthy … candidate BP11 for the treatment of chronic spontaneous or … , a separate Phase 3 trial in asthma patients is being …
Read also: Aurobindo Pharma bags USFDA okay for Duchenne muscular dystrophy drug Deflazacort
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FDA Action Alert: Orchard, ITF, Merck and More
18 Mar 2024 06:20 GMT
… S. subsidiary of Italian pharma Italfarmaco Group, is proposing … for the treatment of Duchenne muscular dystrophy (DMD). The FDA’s verdict … Expanded Label for Cholesterol Drugs
Esperion is proposing to … bispecific antibody authorized to treat both FL and DLBCL. …
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Iran's Pharmaceutical Industry In ‘Crisis’
17 Mar 2024 23:56 GMT
… , raising widespread concerns among pharmaceutical companies.
Local media reports … common medicines and crucial drugs essential for hospitals amid … the nation's muscular dystrophy support association cautioned in December … to complimentary medical treatment.
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Mechanism of Action of SAT-3247 in Muscular Dystrophy: Frank Gleeson
14 Mar 2024 15:13 GMT
WATCH TIME: 3 minutes
"There are stem cells, we discovered, do not properly divide the way they are intended to divide to create new muscle cells. As a result, we believe that the progressive damage to the muscle that is seen in muscular dystrophy is …
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Failure of ALS drug puts a spotlight on controversial FDA approvals
16 Mar 2024 10:13 GMT
… of their treatment. Instead, the trial showed that their drug, Relyvrio, … , Mass.-based Amylyx Pharmaceuticals said last week they … Alzheimer’s and Duchenne muscular dystrophy, a fatal disease that … good treatments. The FDA had approved just two other drugs for …
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Dr Ben Rome Scrutinizes the FDA’s Accelerated Approval Pathway for DMD Treatments, Calls for Policy Reform
15 Mar 2024 21:11 GMT
… moved 5 genetically targeted treatments for Duchenne muscular dystrophy (DMD) through … at a group of drugs that treats DMD, which is … accelerated approval, the trial for that drug—that is presumably … the FDA is requiring that the trial to confirm the drugs’ …
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Türkiye should start financing health, not illness
18 Mar 2024 23:17 GMT
… for all diabetes-related treatments. This negligence is not … its health care and pharma expenses will not … muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), and idiopathic pulmonary … nation's protective pharmaceutical exchange rate policy and …