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A study appearing in Frontiers of Medicine has uncovered a critical role of the BOLA3 gene and its exon 3 inclusion, which is promoted by the heterogeneous nuclear ribonucleoprotein C (HNRNPC), in ...

Riding recent momentum in the Duchenne muscular dystrophy space, Capricor Therapeutics, Wave Life Sciences, Regenxbio and ...

Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is ...

Entrada plans to initiate clinical testing of ENTR-601-44 in Duchenne MD patients amenable to exon 44 skipping later this year.

Announcing a new article publication for Cardiovascular Innovations and Applications journal. Alternative splicing (AS), a critical process for gene expression regulation, allows a single precursor ...

Sionna Therapeutics has closed its initial public offering, raising $191 million for its pipeline of therapies for cystic ...

Computational prediction of gene structure is crucial for interpreting ... Expression-based systems that use only native alignments tend to produce exon-intron structures that are quite accurate.

which chief executive Doug Ingram said showed that the gene therapy was not cannibalising sales of its exon-skipping drugs. Ingram also said that Sarepta was making good progress with other areas ...