A PepGen drug in development for myotonic dystrophy type 1 (DM1) has encouraging early human data that suggest it could be a better treatment than other medicines in clinical testing for this rare, ...

The former Little Mix star recently revealed her twin baby daughters have been diagnosed with SMA.

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 ...

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

Dyne Therapeutics, Inc. develops treatments for muscle diseases using its FORCE platform, with promising candidates DYNE-101 and DYNE-251 targeting DM1 and DMD, respectively. DYNE-251 shows ...

In a review published in the International Journal of Molecular Science, researchers set out to assess how taking magnesium supplements affected skeletal muscle health, with a focus on neuromuscular ...

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

- 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 ...

I've been blogging for a few years about misophonia, an auditory/neurological disorder that I have advocated for because I and (more importantly) one of my adult children are affected. Recently, at ...