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Spinal muscular atrophy: searching for a cure
Organoids with SMA-pathology uncovered key findings about the disease, which could be utilised to develop new therapeutic options.
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spinal muscular atrophy (SMA)
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Truncated version of the SMN2 gene could revolutionise drug discovery
A condensed version of the SMN2 gene could improve discovery of potential therapies for spinal muscular atrophy and other conditions.
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Model for spinal muscular atrophy using human iPSCs
A 2D neuromuscular junction model enables high-throughput screening to discover new treatments for neuromuscular diseases.
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Providing new hope for rare disease using non-viral gene therapy
Viral vectors to deliver gene therapies are utilised by clinically approved therapies. However, this method is not the only option for advanced therapeutics. In this piece, Brent Warner, President, Gene Therapy at Poseida Therapeutics, discusses the balance between efficacy and safety in gene therapy, highlighting positive pre-clinical data for non-viral…
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Challenges and opportunities in gene therapy development
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.
news
Gene found to protect motor neurons against degeneration
Results of an international collaboration show promise for the future of motor neurone disease treatment efforts, as protective gene is identified.
news
New angle of attack on spinal muscular atrophy (SMA)
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.
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JPND launches €23 million call for neurodegenerative disease research
Neurodegenerative diseases such as Alzheimer’s disease and Parkinson’s disease are a global health, economic and social emergency.