Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for ...

Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for myotonic dystrophy type 1 (DM1), the most common adult-onset form of ...

Amy Moffatt's four-year-old son, Oakley, has the most serious form of Spinal Muscular Atrophy (SMA), which causes progressive ...

Repeated exercise, or wasting, can change the way key genes work.

A MUM, whose eight-year-old son has Spinal Muscular Atrophy (SMA), says families have been ‘ignored for years’ as singer Jesy Nelson calls ...

SMA patients in Kerala urge government to allocate funds for free drug distribution to combat the rare genetic disorder.

Biogen has secured European regulatory approval for a high-dose regimen of its Spinraza drug for the neuromuscular disease spinal muscular atrophy, or SMA. Biogen on Monday said the European ...

Some secrets won't stay buried. In this irresistibly twisty drama, a suburban garden club’s perfect world starts to crumble ...

I don't think I'll ever get over it or accept it, but all I can do is try and do my best and try and make change." Although the babies have undergone gene therapy, the 34-year-old star explained the ...

The former Little Mix singer posted an emotional video to Instagram on Sunday, vowing that the girls will "fight all the odds ...

The Little Mix singer, 34, revealed her eight-month-old daughters suffer from a severe muscular disease and will likely be ...

A mother whose son was diagnosed with spinal muscular atrophy (SMA) when he was ten days old has urged singer Jesy Nelson to ...