Duchenne muscular dystrophy Archives -

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Market Access/ News/ News/ Press Releases

Solid Biosciences acquires AavantiBio, conducts $75 million private placement

Nicole Raleigh AAV, aavantbio, acquisition, BAG3, DMD, Duchenne muscular dystrophy, Friedrich's ataxia, gene therapy, M&A, merger, Solid Biosciences 0 Comment

The life sciences company Solid Biosciences is merging with the privately-held gene therapy company AavantiBio in a deal

Solid Biosciences acquires AavantiBio, conducts $75 million private placement

https://pharmaphorum.com/news/solid-biosciences-acquires-aavantibio-conducts-75-million-private-placement/

Market Access/ News/ News/ Press Releases

Translarna not approved by NICE for DMD on NHS

Nicole Raleigh ataluren, DMD, Duchenne muscular dystrophy, NICE, Translarna 0 Comment

In draft guidance published on Friday, it emerged NICE does not recommend PTC Therapeutics’ Translarna (ataluren) for the

Translarna not approved by NICE for DMD on NHS

https://pharmaphorum.com/news/translarna-not-approved-by-nice-for-dmd-on-nhs/

Disease Spotlight/ DMD Main/ News/ News/ R&D

Sarepta files Duchenne muscular dystrophy gene therapy with FDA

Phil Taylor accelerated approval, DMD, Duchenne muscular dystrophy, Sarepta 0 Comment

Sarepta Therapeutics has followed through on its promise to file for accelerated approval of its gene therapy SRP-9001

Sarepta files Duchenne muscular dystrophy gene therapy with FDA

https://pharmaphorum.com/news/sarepta-files-duchenne-muscular-dystrophy-gene-therapy-with-fda/

Young man in a wheelchair looking at the nature

Market Access/ News/ News

Sarepta says early filing for DMD gene therapy is back on

Phil Taylor Duchenne muscular dystrophy, gene therapy, genetic diseases, Sarepta 0 Comment

Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its

Sarepta says early filing for DMD gene therapy is back on

https://pharmaphorum.com/news/sarepta-says-early-filing-for-dmd-gene-therapy-is-back-on/

News/ News/ R&D

Myocarditis case mars Sarepta DMD gene therapy readout

Phil Taylor Duchenne muscular dystrophy, gene therapy, Pfizer, Sarepta, Solid Biosciences 0 Comment

New data on Sarepta’s gene therapy for Duchenne muscular dystrophy (DMD) has gone a long way towards building

Myocarditis case mars Sarepta DMD gene therapy readout

https://pharmaphorum.com/news/myocarditis-case-mars-sarepta-dmd-gene-therapy-readout/

News/ News/ R&D

Sarepta shares yo-yo as FDA places hold on Duchenne trial

Phil Taylor Duchenne muscular dystrophy, Exondys 51, fda, rare disease, regulatory, safety, Sarepta, vesleteplirsen 0 Comment

Shares in Sarepta came under pressure after the biotech said it had temporarily halted a clinical trial of

Sarepta shares yo-yo as FDA places hold on Duchenne trial

https://pharmaphorum.com/news/sarepta-shares-yo-yo-as-fda-places-hold-on-duchenne-trial/

News/ News/ R&D

Astellas takes $170m charge as it drops DMD gene therapies

Phil Taylor Astellas, Duchenne muscular dystrophy, gene therapy, safety 0 Comment

Astellas has said it will book a $170 million impairment charge in its fourth quarter results as a

Astellas takes $170m charge as it drops DMD gene therapies

https://pharmaphorum.com/news/astellas-takes-170m-charge-as-it-drops-dmd-gene-therapies/

News/ News/ R&D

Sarepta DMD rival PepGen files $115m IPO

Phil Taylor Duchenne muscular dystrophy, financing, NS Pharma, Oligonucleotide, PepGen, Sarepta 0 Comment

Just days after dosing its first patient in a Duchenne muscular dystrophy trial, US biotech PepGen has pushed

Sarepta DMD rival PepGen files $115m IPO

https://pharmaphorum.com/news/sarepta-dmd-rival-pepgen-files-115m-ipo/

News/ News/ R&D

Sarepta says early filing for DMD gene therapy may be back on

Phil Taylor Duchenne muscular dystrophy, gene therapy, Pfizer, rare disease, Sarepta, Solid Biosciences 0 Comment

The development of gene therapies for Duchenne muscular dystrophy has proved to be challenging, but one of the

Sarepta says early filing for DMD gene therapy may be back on

https://pharmaphorum.com/news/sarepta-says-early-filing-for-dmd-gene-therapy-may-be-back-on/

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