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Duchenne Muscular Dystrophy

Showing 15 posts of 34 posts found.

NICE issues positive final guidance for treatment of Duchenne Muscular Dystrophy from Santhera

January 29, 2025
Sales 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 …

News

Santhera Pharmaceuticals announces LIONHEART study results for corticosteroid vamorolone

October 1, 2024
Research 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 …

News

Regenxbio announces initial efficacy data from phase 1/2 AFFINITY DUCHENNE trial

March 6, 2024
Research 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 …

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FDA pre-meeting report raises concerns over Sarepta’s gene therapy for Duchenne muscular dystrophy

May 11, 2023
Medical 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 …

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FDA approves NS Pharma’s Viltepso for Duchenne muscular dystrophy, but concerns over benefit persist

August 13, 2020
Sales 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 …

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Roche terminates Duchenne muscular dystrophy candidate due to poor clinical outlook

November 8, 2019
Medical 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 …

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shutterstock_159488225

Capricor’s share price rockets up by 150% on Phase 2 Duchenne muscular dystrophy success

July 16, 2019
Manufacturing 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 …

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shutterstock_36423250

Summit Pharma axes Duchenne drug after Phase 2 failures

June 28, 2018
Research 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 …

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shutterstock_159488225

Sarepta shares surge after gene therapy leads to microdystrophin expression in DMD patients

June 20, 2018
Research 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 …

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Top Ten most popular articles on Pharmafile.com this week

February 2, 2018
Business 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 …

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accesstoraxone

Santhera’s Raxone rejected in Europe for Duchenne muscular dystrophy

January 29, 2018
Sales 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, …

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Santhera ‘surprised’ by European rejection of DMD drug

September 18, 2017
Sales 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 …

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golden_retriever

Breakthrough gene therapy shows promise in Duchenne muscular dystrophy

July 26, 2017
Research 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 …

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thumbs_up

Duchenne drug made available to UK patients early

June 27, 2017
Manufacturing 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 …

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money_packets

Duchenne drug poised to cost $35,000 but with major catch

May 9, 2017
Manufacturing 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 …

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