Despite the promise of gene therapies, significant challenges have emerged in the field. Dr Carsten Brunn discusses the current obstacles and opportunities when developing gene therapies.
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spinal muscular atrophy (SMA)
Results of an international collaboration show promise for the future of motor neurone disease treatment efforts, as protective gene is identified.
Spinal muscular atrophy (SMA) in its most severe form is incurable and fatal in early childhood, but now researchers are mounting a multi-pronged attack for patients and their families.
Neurodegenerative diseases such as Alzheimer’s disease and Parkinson’s disease are a global health, economic and social emergency.