Duchenne Muscular Dystrophy
NICE issues positive final guidance for treatment of Duchenne Muscular Dystrophy from Santhera
January 29, 2025Sales and Marketing Duchenne Muscular Dystrophy, EC, EMA, FDA, Santhera Pharmaceuticals, positive
Santhera Pharmaceuticals has received positive final guidance from the National Institute for Care and Excellence (NICE) for AGAMREE (vamorolone) to …
Santhera Pharmaceuticals announces LIONHEART study results for corticosteroid vamorolone
October 1, 2024Research and Development Cardiology, Duchenne Muscular Dystrophy, Immunology, Santhera Pharmaceuticals, clinical studies
Santhera Pharma has announced the results of its LIONHEART study, which evaluated vamorolone – a corticosteroid – and confirmed its …
Regenxbio announces initial efficacy data from phase 1/2 AFFINITY DUCHENNE trial
March 6, 2024Research and Development Duchenne Muscular Dystrophy, Musculo-skeletal disorder, Regenxbio, clinical trial
Regenxbio has announced additional interim safety data from its phase 1/2 AFFINITY DUCHENNE trial of RGX-202 in patients with Duchenne …
FDA pre-meeting report raises concerns over Sarepta’s gene therapy for Duchenne muscular dystrophy
May 11, 2023Medical Communications Duchenne Muscular Dystrophy, FDA, Musculo-skeletal disorder, Sarepta Therapeutics, gene therapy
The US Food and Drug Administration (FDA) has released a report in preparation for its 12 May decision on Sarepta …
FDA approves NS Pharma’s Viltepso for Duchenne muscular dystrophy, but concerns over benefit persist
August 13, 2020Sales and Marketing Duchenne Muscular Dystrophy, FDA, NS pharma, Viltepso
NS Pharma has claimed a tentative FDA approval for its Viltepso (viltolarsen) injection in the treatment of Duchenne muscular dystrophy …
Roche terminates Duchenne muscular dystrophy candidate due to poor clinical outlook
November 8, 2019Medical Communications, Research and Development Duchenne Muscular Dystrophy, Roche, pharma
Roche has revealed its decision to terminate further development of its anti-myostatin adnectin protein agent RG6206 after it became apparent …
Capricor’s share price rockets up by 150% on Phase 2 Duchenne muscular dystrophy success
July 16, 2019Manufacturing and Production, Research and Development CAP-1002, Capricor Therapeutics, Duchenne Muscular Dystrophy, pharma
Capricor Therapeutics has unveiled impactful new Phase 2 data for its investigational allogeneic cardiosphere-derived cell therapy CAP-1002 in the treatment …
Summit Pharma axes Duchenne drug after Phase 2 failures
June 28, 2018Research and Development Duchenne Muscular Dystrophy, Summit Pharmaceuticals, pharma, trial failure
Summit Therapeutics has revealed it is to terminate development of its Duchenne muscular dystrophy (DMD) drug ezutromid after it failed …
Sarepta shares surge after gene therapy leads to microdystrophin expression in DMD patients
June 20, 2018Research and Development Duchenne Muscular Dystrophy, pharma, sarepta
This week, shares in Sarepta Therapeutics rocketed by 80% after the company released preliminary data from a Phase 1/2a trial …
Top Ten most popular articles on Pharmafile.com this week
February 2, 2018Business Services, Manufacturing and Production, Medical Communications, Research and Development, Sales and Marketing Ablynx, Alzheimer's, Apotex, AstraZeneca, Brexit Health Alliance, CDC, Duchenne Muscular Dystrophy, EMA, Italy, Sanofi, amazon, brexit, santhera, top ten
It’s the first Friday of February, and it’s time for another recap of the top ten articles most popular with …
Santhera’s Raxone rejected in Europe for Duchenne muscular dystrophy
January 29, 2018Sales and Marketing Duchenne Muscular Dystrophy, EMA, Europe Santhera, pharma
While the EMA’s Committee for Medicinal products for Human Use (CHMP) recommended a spree of treatments over the past week, …
Santhera ‘surprised’ by European rejection of DMD drug
September 18, 2017Sales and Marketing Duchenne Muscular Dystrophy, biotech, drugs, pharma, pharmaceutical, santhera
Santhera Pharmaceutical has announced the EMA’s CHMP rejected its treatment for Duchenne muscular dystrophy (DMD) but revealed that it planned …
Breakthrough gene therapy shows promise in Duchenne muscular dystrophy
July 26, 2017Research and Development DMD, Duchenne Muscular Dystrophy, University of London, University of Nantes, biotech, drugs, pharma, pharmaceutical
New research, led by teams from AFM-Telethon laboratory in Nantes and the University of London, have developed a gene therapy …
Duchenne drug made available to UK patients early
June 27, 2017Manufacturing and Production, Sales and Marketing Duchenne Muscular Dystrophy, Raxone
UK patients will be given access to Santhera Pharmaceutical’s Duchene Muscular Dystrophy (DMD) drug, Raxone (idebenone), through the Early Access …
Duchenne drug poised to cost $35,000 but with major catch
May 9, 2017Manufacturing and Production, Sales and Marketing Duchenne Muscular Dystrophy, PTC Therapeutics, marathon
For those that have not followed the story, Emflaza was successfully taken through the FDA regulatory process by Marathon as …
Latest content
FibroFind appoints Lee Borthwick as CEO
