News & Views: Scientists developed a combined stem-cell and genetherapy strategy for ALS. Hideyuki Okano discusses the first clinical results, which will inform further clinical trials of this neurodegenerative disease. MIT mcgovernmit KeioGlobal
Amyotrophic lateral sclerosis is a devastating neurodegenerative disease with no curative treatment. Riluzole and edaravone are approved as anti-ALS drugs, but only modestly slow disease progression.
Therefore, new treatments are being developed, not only with small chemical compounds but also with new drug-discovery modalities such as biologics , regenerative medicines, gene therapies, gene editing technologies, antisense oligonucleotides and mRNA-based medicines
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