Congenital muscular dystrophies (CMDs) comprise a heterogeneous group of inherited disorders primarily affecting muscle integrity and function, often with accompanying brain and ocular abnormalities.

An HIV-derived nucleoside therapy now treats rare genetic diseases by restoring mitochondrial DNA and improving muscle ...

On January 4, former Little Mix singer Jesy Nelson took to Instagram to reveal that her twins have been diagnosed with spinal ...

People frequently wait years to be "taken seriously," postcode lotteries for access to care and a general lack of knowledge are among some of the key findings from a University of Aberdeen ...

Former Little Mix star breaks down as she explains eight-month-old Ocean and Story have severe SMA and may never walk ...

A team at the Centro Nacional de Investigaciones Cardiovasculares (CNIC) has developed an innovative method known as TEVs-TTN, for studying the specific mechanical functions of proteins through their ...

A study of adults aged 60 years and older found that the use of statin therapy as primary cardiovascular disease (CVD) prevention was effective for preventing CVD and all-cause mortality, even in ...

Singer Jesy Nelson has pledged to "fight as much as I can" to get all babies tested for a rare muscle disease at birth, after ...

The BBC's Ben Morris describes living with spinal musclar atrophy, after Little Mix's Jessy Nelson spoke about it.

Jesy Nelson revealed in an Instagram video on Sunday, Jan. 4, that her twin daughters have been diagnosed with Spinal ...