Advocacy

Leading advocates and experts share their experiences
Jan-Anders Karlsson on Working with the Cystic Fibrosis Trust

Jan-Anders Karlsson on Working with the Cystic Fibrosis Trust

https://www.youtube.com/watch?v=Ls0lzSOuhIM   Jan-Anders Karlsson discusses Verona Pharma's lead candidate, RPL554, as a potential therapy for cystic fibrosis (CF) patients and his company's work with theCystic Fibrosis Trust. The Cystic Fibrosis Trust is the...

Summit Therapeutics Discusses It’s Multiple DMD Partnerships

Summit Therapeutics Discusses It’s Multiple DMD Partnerships

https://www.youtube.com/watch?v=WiK5qFBg6ro Glyn Edwards of Summit Therapeutics discusses his company's partnerships with the Duchenne muscular dystrophy (DMD) community and with Sarepta Therapeutics. DMD is one of the most common, fatal genetic disorders diagnosed in...

Being Diagnosed with Charcot-Marie-Tooth Disease

Being Diagnosed with Charcot-Marie-Tooth Disease

Charcot-Marie-Tooth disease (CMT) is one of the most common inherited neurological disorders, affecting approximately 1 in 2,500 people in the United States. The disease is named for the three physicians who first identified it in 1886 - Jean-Martin Charcot and Pierre...

Thijs Spoor of AzurRx Provides an Overview of Cystic Fibrosis

Thijs Spoor of AzurRx Provides an Overview of Cystic Fibrosis

Thijs Spoor of AzurRx Provides an Overview of Cystic Fibrosis Cystic fibrosis (CF) is a life-threatening genetic disease that primarily affects the lungs and digestive system. An estimated 30,000 children and adults in the United States (70,000 worldwide) have CF. In...

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