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Sarepta dives deeper into gene therapy
https://pharmaphorum.com/news/sarepta-dives-deeper-gene-therapy/
DMD
An experimental gene therapy for DMD shows positive results
Cell & Gene Therapy 2018
Living with DMD: Ensuring equal access to quality of life
Making sure everyone with DMD gets access to treatments and the ‘essential’ interventions that can make life worth living is key.
Accelerating access for rare disease patients – the need to harmonise Europe’s regulation
Santhera is putting patient needs at the heart of its rare disease speciality business. Chief Executive Officer Thomas Meier, PhD, talks about the company’s mission – and how rationalising Europe’s drug review systems could help patients.
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