In Study 041, Translarna showed a significant improvement in six key study endpoints compared with placebo after 72 weeks. The Food and Drug Administration (FDA) has accepted for review the ...
Drs. Sharon and Aroya will enrich our knowledge on the efficacy of safety ADAR-based RNA editing, a first step towards RNA editing treatment in CHM patients.” — Neal Bench, CRF board president ...
WATERTOWN, Mass., May 24, 2023 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today announced that the company ...
ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, today announced the close of over $29 ...
Premature termination codons (PTCs) account for 10 to 20% of genetic diseases in humans. The gene inactivation resulting from PTCs can be counteracted by the use of drugs stimulating PTC readthrough, ...
BETHESDA, Md.--(BUSINESS WIRE)--Today, the Cystic Fibrosis Foundation announced an additional investment of up to $24 million in Prime Medicine to continue the development of a gene editing therapy ...
In a global first, scientists have used advanced base editing to repair a deadly gene mutation in a newborn, marking a major leap forward in the treatment of rare ...
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