The Duchenne muscular dystrophy (DMD) landscape across the seven major markets (7MM) - the US, France, Germany, Italy, Spain, ...

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 ...

Data from DELIVER and ACHIEVE Clinicals Trial to be Presented - - Presentation on ACHIEVE Trial in DM1 and Company Symposium to Feature Data on the Use of Splicing Correction as a Prognostic Biomarker ...

Managed Healthcare Executive provides C-suite executives at health plans and provider organizations with news, analysis, ...

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

The drug – formerly known as SRP-5051 – is a more potent version of Exondys 51 (eteplirsen), an exon-skipping therapy that can be used to treat around 13% of the population with the muscle ...

Intronic primers flanking exon 9 were designed using the PAC sequence available in the Genbank sequence database (clone DJ0054D12; accession number AC006342). PCR amplification of an approximate 6 ...

Entrada Therapeutics has secured authorisation from the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) to ...

Halozyme (HALO) announced that Janssen-Cilag International, a Johnson & Johnson (JNJ) company, received a positive opinion from the ...