Solid Biosciences’ gene therapy uses a shortened version of the dystrophin protein, which is engineered to mimic the key ...

Medically reviewed by Smita Patel, DO Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed ...

A new study from the University of Ottawa’s Faculty of Medicine sheds light on a potential breakthrough in treating muscle ...

Utrophin increase in muscle cells after transcriptional adaptation normalises cell function in Duchenne muscular dystrophy ...

the patient-derived heart organoids attained the rhythmic activity seen in healthy organoids. Similar results were observed with an ASO targeting a different region of dystrophin that is currently ...

DMD is an X-linked disorder characterized by progressive muscle weakness and wasting due to the absence of a protein called dystrophin, which in turn causes degeneration of skeletal and cardiac muscle ...

Muscular dystrophy is a disease caused by a genetic mutation. The muscles of muscular dystrophy patients weaken.. | Genetics ...

SAST and syntrophin were highly associated with purified microtubules and microtubule-associated proteins, whereas utrophin and dystrophin were only partially associated with microtubules.

Solid Biosciences' SGT-003 gene therapy showed strong dystrophin expression, muscle health improvements, and no serious safety concerns in early trial data.

Duchenne muscular dystrophy (DMD) is a rare hereditary disease that is associated with progressive muscle wasting. The ...

HOUSTON--(BUSINESS WIRE)--IPS HEART has been awarded Orphan Drug Designation (ODD) by the FDA for GIVI-MPCs based on its unique ability to create new muscle with full length dystrophin in Becker ...

This is due to a fault in the gene producing dystrophin. Muscles to become weaker ... ability to breathe as well as the function of the heart, as the heart is a muscle too. The missing protein ...