Rare diseases

News

Minister says using generic CF drug is an option in Orkambi ...

Vertex is coming under increased pressure to sign a deal with the NHS covering its Orkambi cystic fibrosis (CF) drug, with a government minister suggesting she would consider implementing l

Market Access

pharmaphorum podcast bluebird bio Nicola Redfern

Gene therapy and market access: the pharmaphorum podcast

For episode 10 of the pharmaphorum podcast I spoke to Nicola Redfern, who’s general manager at bluebird bio in the UK.

News

engineering and genetic editing through the Crispr technique. DNA chain breaking down. 3D rendering

Amicus expands gene therapy collaboration

Amicus Therapeutics and the Perelman School of Medicine at the University of Pennsylvania have announced a major expansion to their R&D collaboration for gene therapies.

News

Novartis shows off pipeline - including 25 potential blockbu...

Novartis has showcased its pipeline at its annual meet-the-management day, where investors and analysts got to hear company leaders give an overview of its drug pipeline.

Events

Partner Content

SMi's 9th Annual Orphan Drugs & Rare Diseases Conference

Orphan Drugs & Rare Diseases

News

NHS chief hails SMA drug deal - and was that a swipe at Vert...

Children in England with the rare muscle wasting disease spinal muscular atrophy (SMA) will get access to Biogen’s super-expensive new drug Spinraza after the manufacturer struck a deal wit

Minister says using generic CF drug is an option in Orkambi ...

Gene therapy and market access: the pharmaphorum podcast

Amicus expands gene therapy collaboration

Novartis shows off pipeline - including 25 potential blockbu...

SMi's 9th Annual Orphan Drugs & Rare Diseases Conference

NHS chief hails SMA drug deal - and was that a swipe at Vert...

The long-delayed promise of the microbiome, with Sam Possemi...

How to meet your physicians where they are online

Rare diseases

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