Locanabio Presents Preclinical Data from its Vectorized snRNA Exon Skipping Program…
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Browsing Tag
C9orf72
Wave Life Sciences Announces Publication of Foundational Preclinical Data Supporting…
Preclinical data published in Molecular Therapy Nucleic Acids demonstrate that WVE-004 potently reduces C9orf72 transcriptional variants and poly(GP) dipeptide repeat proteins in mice for…
Biogen and Ionis Announce Topline Phase 1 Study Results of Investigational Drug in…
BIIB078, an investigational antisense oligonucleotide for C9orf72-associated amyotrophic lateral sclerosis (ALS), did not show clinical benefit; clinical program will be discontinuedBiogen…
Discovery of the role of a key gene in the development of ALS: Loss of function of…
Amyotrophic lateral sclerosis, or ALS, attacks nerve cells known as motor neurons in the brain and spinal cord, gradually leading to paralysis. The loss of function of an important gene,…