Multidisciplinary coordination across prescribing teams, nursing, laboratory medicine, finance, and infusion centers is crucial for gene therapy delivery in Duchenne muscular dystrophy (DMD).

Marcos Reyes and Valeria Martínez are in a tough spot, unsure which of their twins should receive the treatment they ...

U.K. regulators cleared a multiple myeloma drug GSK withdrew from market three years ago. Elsewhere, an investment firm pressed Elevation Oncology to liquidate and the FDA relaxed risk monitoring ...

Just a few weeks after closing a $155 million series B, RNA editing biotech AIRNA is bringing an experienced gene therapy ...

Sarepta Therapeutics SRPT announced updates from its clinical programs, which focus on targeting different subtypes of limb-girdle muscular dystrophy (LGMD), a group of rare genetic disorders marked ...

The life sciences hedge fund was down 32 percent in the first quarter alone in a generally rough period for much of the ...

A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, but researchers say the lessons learned could pave the way for better, more effective treatments.

The law firm of Kessler Topaz Meltzer & Check, LLP ( is currently investigating potential violations of the federal ...