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In draft guidance published on Friday, it emerged NICE does not recommend PTC Therapeutics’ Translarna (ataluren) for the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

Disease Spotlight/ DMD Main/ Views and analysis

Curing DMD: Travelling together to find a meaningful destination

Amanda Barrell disease, DMD, Duchenne muscular dystrophy, Duchenne Parent Project Spain, Joaquim Brites, Marisol Montolio 0 Comment

For many years, Duchenne muscular dystrophy (DMD) was something of a forgotten disease. The boys, and some girls,

Curing DMD: Travelling together to find a meaningful destination

https://pharmaphorum.com/disease-spotlight/dmd-main/curing-dmd-travelling-together-to-find-a-meaningful-destination/

Growing concept eco Group hand children planting together on soil backgroud

Disease Spotlight/ DMD Main/ Views and analysis

Increase collaboration to widen access to DMD drugs, trials and care

louise.bellamy@pharmaphorum.com access to clinical trials, access to drugs, DMD, Duchenne muscular dystrophy 0 Comment

When it comes to fighting a rare disease like Duchenne muscular dystrophy (DMD), it’s a matter of strength

Increase collaboration to widen access to DMD drugs, trials and care

https://pharmaphorum.com/disease-spotlight/increase-collaboration-to-widen-access-to-dmd-drugs-trials-and-care/

Market Access/ Patients/ Views & Analysis/ Views & Analysis/ Views and analysis

Collaboration can ensure access to life-changing innovation

Amanda Barrell DMD, multiple sclerosis, NICE, QALY, risk sharing scheme 0 Comment

As a pioneering risk sharing scheme has shown, new medications can be the catalyst for a whole new

Collaboration can ensure access to life-changing innovation

https://pharmaphorum.com/views-analysis-market-access/access-life-changing-innovation-risk-sharing-scheme/

Disease Spotlight/ DMD Main/ Views and analysis

DMD campaigners: Advocating for change

Dominic Tyer disease, DMD, DMD campaigners, DMD campaigning, Duchenne, Klair Bayley, Parent Project Muscular Dystrophy, Pat Furlong, Ricky Andreis, Santhera, Save Our Sons, Swedish Muscular Dystrophy Research Foundation 0 Comment

The internet and social media are often heralded as a panacea for rare disease patients and their carers

DMD campaigners: Advocating for change

https://pharmaphorum.com/disease-spotlight/dmd-main/dmd-campaigners-advocating-change/

News/ Pfizer/ Rare diseases

Pfizer axes development of Duchenne muscular dystrophy antibody

Piotr Wnuk DMD, Duchenne, Duchenne muscular dystrophy, Pfizer, rare disease 0 Comment

Pfizer has axed trials of its domagrozumab antibody for Duchenne muscular dystrophy (DMD), after it failed against efficacy

Pfizer axes development of Duchenne muscular dystrophy antibody

https://pharmaphorum.com/news/duchenne-trials-axed-pfizer-dmd/

Market Access/ News/ News/ News/ R&D

Sarepta dives deeper into gene therapy

Piotr Wnuk DMD, Duchenne muscular dystrophy, Lacerta Therapeutics, Sarepta 0 Comment

Sarepta Therapeutics has handed $30 million to Lacerta in exchange for a group of gene therapies, including a

Sarepta dives deeper into gene therapy

https://pharmaphorum.com/news/sarepta-dives-deeper-gene-therapy/

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