With a tank of zebrafish, Queensland researcher Jean Giacomotto was able to test a never-before seen genetic variant ...

A couple whose son died from a rare genetic condition are encouraging others to take part in screening which they say could ...

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...

More than two years after the Government committed to adding SMA to newborn testing, families are still waiting ...

Findings from a new study in the Journal of Neuromuscular Diseases, published by IOS Press, demonstrate the effectiveness of disease-modifying treatments (DMTs) in infants with spinal muscular atrophy ...

Newborn babies in Hull will be able to be screened for more than 200 rare genetic conditions as part of a national study to ...

Spinal muscular atrophy is a debilitating genetic condition that’s usually fatal by a few years of age. But an intriguing case study might demonstrate a simple new treatment, with a child showing no ...

What is SMA? The rare genetic condition that Jesy Nelson’s twins have - Former Little Mix star revealed her twin daughters had been diagnosed with Spinal Muscular Atrophy (SMA) Type 1 ...

The U.S. Food and Drug Administration has approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of spinal muscular atrophy (SMA) in patients 2 years and older with confirmed mutation in ...

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com. Topline results from a phase 3 clinical trial showed an ...

Genethon highlights latest developments in gene therapies for diseases once considered incurable, including Duchenne muscular dystrophy ...