Leni Forrester, 2, has Sanfilippo Syndrome, a rare genetic condition often described as childhood dementia - and popstar Jesy ...

Scotland launches a newborn screening pilot for spinal muscular atrophy (SMA) via the heel prick test in Glasgow, aiming for ...

The Little Mix star has been campaigning for SMA to be a part of the standard screening for babies after birth ...

More than 100 Southern Californians will gather Saturday, February 28, at A Snail's Pace running shop in Brea for a family-friendly Rare Disease Day 5K fun run and walk, rallying support for the more ...

Aardvark Therapeutics Inc. AARD on Monday announced to voluntarily pause its Phase 3 HERO trial for Prader-Willi Syndrome. The update has sent the stock plunge almost 61%. The decision comes as the ...

A YOUNGSTER who has been diagnosed with a one in a million condition hasn’t let it hold him back and he had the ch... Local ...

The Little Mix star, 34, welcomed her daughters Ocean and Story in 2025, but in January she shared both babies had been ...

Jesy Nelson looked incredible in a crop top as she got back to work filming for her new TV documentary amid her baby twins' ...

Jesy Nelson has hit out in a social media rant in which she slams women who objectify and scrutinise other women's bodies. The mum-of-two, who recently announced that her baby twin girls have SMA, ...

The star has kept fans updated on her twins' health on social media ...

A woman who had to STAND to eat and drink due to a rare condition has had life-changing surgery so she can enjoy her favourite foods again – after shrinking to six stone. Elise Banyard, 26, a care ...

Eliana Merle Barclays Bank PLC, Research Division. Great. Good afternoon, everyone. I'm Ellie Merle, one of the biotech analysts here at Barclays. Very happy to have Ionis here wi ...