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 ...

U.S. Health Secretary Robert F. Kennedy Jr. on Tuesday added two rare genetic disorders, Duchenne Muscular Dystrophy and ...

The UAE has become the second country worldwide to approve Itvisma, a groundbreaking gene therapy for spinal muscular atrophy ...

Grants include research funding in amyotrophic lateral sclerosis (ALS), Charcot-Marie-Tooth disease (CMT), Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy (FSHD), mitochondrial ...

I don’t want to be stuck in a box,” says a Tauranga woman suffering from a hereditary muscular disorder. “Stuck in a box” is ...

Inspired by his father, Grandview High School student Josue Clara started a nonprofit called Beyond Rare to build awareness ...

Aging taps us on the shoulder in many ways: wrinkles, thinning hair, loss of flexibility, slowing of the brain. But the ...

Regulator approves Itvisma for adults and children aged two and above, positioning the UAE among the first countries globally to authorise the treatment for a wider age group ...

Dubai, UAE: The Emirates Drug Establishment (EDE) has announced the approval of Itvisma™ (onasemnogene abeparvovec), a gene therapy that uses an adeno-associated viral vector to treat spinal muscular ...

A review on autoimmune neurological diseases reveals what occurs in our body when the immune system, by mistake, produces antibodies that target a protein essential ...

The Emirates Drug Establishment (EDE) has announced the approval of Itvisma (onasemnogene abeparvovec), a gene therapy that ...

The treatment targets and stops production of abnormal huntingtin proteins responsible for disease progression. Delivered ...