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Top Bulk Deflazacort Powder Manufacturers in China PharmaSources.com
April 13, 2021
Deflazacort (C25H31NO6) is a glucocorticoid that works as an immunosuppressant and anti-inflammatory. The FDA (Food and Drug Administration) considers it a first-class medication approved in 2017. Generally, it is a corticosteroid prodrug that can ...
Pfizer enrols first UK participant into global DMD gene therapy study pharmatimes
May 14, 2021
Pfizer has announced that it has enrolled the first UK participant in a global Phase III study aiming to evaluate its investigational gene therapy candidate in boys with Duchenne muscular dystrophy (DMD).
Treatment for rare Duchenne muscular dystrophy mutation approved by FDA europeanpharmaceuticalreview
March 01, 2021
The FDA conditionally approved Sarepta’s Amondys 45 (casimersen) after interim Phase III results indicated the treatment is reasonably likely to be of clinical benefit.
Givinostat from Italfarmaco gets FDA rare pediatric disease designation in duchenne muscular dystrophy expresspharma
October 10, 2020
The Italfarmaco Group provided an update on the development of Givinostat, its proprietary histone deacetylase (HDAC) inhibitor, in boys with Duchenne Muscular Dystrophy (DMD).
Positive results in Duchenne muscular dystrophy (DMD) trial europeanpharmaceuticalreview
October 09, 2020
Interim trial results suggest vamorolone may offer a superior alternative to long-term treatment with high-dose glucocorticoids in young Duchenne muscular dystrophy (DMD) patients.
US FDA approves Viltepso injection for treatment of Duchenne muscular dystrophy mutation expresspharma
August 14, 2020
The US Food and Drug Administration granted accelerated approval to Viltepso (viltolarsen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.
MHRA extends access to Santhera's DMD drug via the EAMS pharmatimes
June 24, 2020
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has renewed for a further year the Early Access to Medicines Scheme (EAMS) scientific opinion for Santhera's Duchenne muscular dystrophy (DMD) drug idebenone.
Catabasis, Duchenne UK announce partnership to evaluate edasalonexent in phase 2 non-ambulatory DMD trial pharmaceutical-business-review
January 13, 2020
Catabasis Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, and Duchenne UK, a charity that seeks to fund and accelerate treatments and a cure for Duchenne muscular dystrophy (DMD), have entered into a partnership for ...
FDA Grants Accelerated Approval to Rare Duchenne Muscular Dystrophy Mutation Treatment americanpharmaceuticalreview
December 16, 2019
The U.S. Food and Drug Administration (FDA) granted accelerated approval to Vyondys 53 (golodirsen) injection to treat Duchenne muscular dystrophy (DMD) patients who have ...
Sarepta’s DMD therapy Vyondys 53 secures FDA accelerated approval pharmaceutical-technology
December 16, 2019
Sarepta Therapeutics has secured accelerated approval from the US Food and Drug Administration (FDA) for Vyondys 53 (golodirsen) to treat duchenne muscular dystrophy (DMD) patients with ...

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