PTC Therapeutics Archives -

Q&A: PTC Therapeutics’ Alberto Vicent on listening to the ultra-rare disease communities

When it comes to ultra-rare diseases, the smaller the patient population, the bigger the challenges. As a global

Listening to ultra-rare disease communities

https://pharmaphorum.com/views-analysis-market-access/listening-to-ultra-rare-disease-communities/

Young man in a wheelchair looking at the nature

Scotland opens door to Translarna for Duchenne muscular dystrophy

Phil Taylor asthma, Duchenne muscular dystrophy, Dupixent, HEOR, PTC Therapeutics, rare disease, Regeneron, Sanofi, Scotland, Translarna 0 Comment

A disparity in UK access to a medicine for Duchenne muscular dystrophy (DMD) has been addressed, after Scotland

Scotland opens door to Translarna for Duchenne muscular dystrophy

https://pharmaphorum.com/news/scotland-opens-door-to-translarna-for-duchenne-muscular-dystrophy/

News/ News/ News/ Patients/ R&D

Sarepta goes up, PTC goes down – DMD research sways the market

Piotr Wnuk DMD, Duchenne muscular dystrophy, neurology, PTC Therapeutics, Rare diseases 0 Comment

US biotech Sarepta’s shares jumped by as much as 60% after a gene therapy produced dramatic results in

Sarepta goes up, PTC goes down – DMD research sways the market

https://pharmaphorum.com/news/dmd-therapy-research-sways-market/

Several Prescription Pill Bottles in a Pile

News/ News/ R&D

PTC shares soar after SMA drug data impresses market

Piotr Wnuk AveXis, Biogen, Novartis, PTC Therapeutics, SMA, spinal muscular atrophy, Spinraza 0 Comment

After failing three times to bring their Duchenne therapy to the US market, PTC Therapeutics might be on

PTC shares soar after SMA drug data impresses market

https://pharmaphorum.com/news/ptc-therapeutics-posts-solid-sma-data/

Market Access/ News/ News/ News/ R&D/ Rare diseases/ UK & Europe

Pharma collaborates to improve chances of NICE backing Duchenne drugs

Richard Staines Duchenne muscular dystrophy, Highly Specialised Technology, Highly Specialised Technology appraisals, Pfizer, PTC Therapeutics, Rare diseases, Roche, Sarepta 0 Comment

Project will initially focus on DMD but could benefit other rare diseases

Pharma collaborates to improve chances of NICE backing Duchenne drugs

https://pharmaphorum.com/news/pharmas-join-improve-duchenne-muscular-dystrophy/

News/ US

PTC Therapeutics fails to convince FDA panel about DMD drug

Richard Staines Duchenne muscular dystrophy, PTC Therapeutics, Rare diseases, Sarepta 0 Comment

FDA advisers have voted against approval for PTC Therapeutics’ Duchenne drug Translarna because of doubts about its efficacy.

PTC Therapeutics fails to convince FDA panel about DMD drug

https://pharmaphorum.com/news/ptc-therapeutics-fails-convince-fda-panel-dmd-drug/

News/ US

FDA reviewers slam PTC’s Duchenne drug ahead of meeting

Richard Staines PTC Therapeutics, Rare diseases, regulation, Sarepta 0 Comment

FDA staff not persuaded by patchy clinical trial evidence for Translarna.

FDA reviewers slam PTC’s Duchenne drug ahead of meeting

https://pharmaphorum.com/news/fda-reviewers-slam-ptc-dmd-drug-ahead-key-meeting/

News/ US

Sarepta announces Allergan veteran as next CEO

Richard Staines Duchenne muscular dystrophy, PTC Therapeutics, Rare diseases, Sarepta 0 Comment

Sarepta has announced former Allergan president Doug Ingram will be its new CEO. Ingram will be the company’s

Sarepta announces Allergan veteran as next CEO

https://pharmaphorum.com/news/sarepta-announces-doug-ingram-new-ceo/

News/ US

Outrage over Duchenne drug price reinflamed by PTC’s $35,000 price tag

Richard Staines drug prices, Duchenne muscular dystrophy, PTC Therapeutics, Sarepta, ultra-orphan, ultra-rare diseases 0 Comment

Families affected by the rare muscle wasting disease Duchenne muscular dystrophy will still have to pay a huge

Outrage over Duchenne drug price reinflamed by PTC’s $35,000 price tag

https://pharmaphorum.com/news/dmd-families-will-still-pay-huge-premium-old-drug-says-ptc/

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