PepGen’s lead candidate for myotonic dystrophy type 1 barely beat the placebo in a Phase 2 trial in terms of fixing incorrect ...

The company discontinued developing emugrobart after two studies showed the drug didn’t achieve intended outcomes.

Muscle ultrasound has emerged as a pivotal, non‐invasive imaging modality that offers real‐time insights into muscle structure and pathology. This technique enables both qualitative and quantitative ...

Genentech is halting development of an antibody for two rare genetic diseases after the candidate failed to boost muscle ...

The big pharma company will no longer progress emugrobart to late-stage trials in FSHD and SMA due to a lack of efficacy.

Biomedical engineers have grown muscles in a lab to better understand and test treatments for a group of extremely rare muscle disorders called dysferlinopathy or limb girdle muscular dystrophies 2B ...

A new method allows large quantities of muscle stem cells to be safely obtained in cell culture. This provides a potential for treating patients with muscle diseases -- and for those who would like to ...

Spinal and Bulbar Muscular Atrophy (SBMA) is a rare inherited disease that causes progressive muscle weakness and wasting in ...

The type 1 ryanodine receptor (RyR1) is an important calcium release channel in skeletal muscles essential for muscle contraction. It mediates calcium release from the sarcoplasmic reticulum, a ...

- All Evaluable Patients in the 3.4 mg/kg Cohort Treated with DYNE-101 Q4W Demonstrated Consistent Splicing Correction with a 19% Mean Improvement Across 22-Gene Panel at 3 Months - - Improvement in ...