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People with disabilities and their families are in the midst of the crisis of their lives, especially in New South Wales.

The new trial aims to reduce frailty and improve mental health by connecting older Australians and preschoolers

Muscular Dystrophy NSW (MDNSW) will be holding its first-ever Big Red Roll + Stroll (BRRS) community event, enabling people living with neu

The national MS Gong Ride goes virtual for the first time in 2020 with thousands of riders raising funds for people living with MS.

Patients with Rett syndrome have similar spinal surgery outcomes to those of people with cerebral palsy (CP), according to new research.

Three grants given researchers at two centres in Australia will advance work into ways of preserving nerve cells in people with ALS

Team Gleason and CureDuchenne have teamed with Google’s Project Euphonia to make automated speech recognition more accessible to people wit

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for ne

Pharnext’s investigational oral combination therapy PXT864 prevents degeneration of motor neurons and their point of contact with...

NS Pharma‘s Viltepso (viltolarsen) is now commercially available in the U.S. for people with Duchenne muscular dystrophy (DMD) amenable to e

The European Commission (EC) approved Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with Kalydeco (ivacaftor) to treat cystic fi

Evrysdi™ (risdiplam) is the first and only oral medication indicated for the treatment of spinal muscular atrophy (SMA) in patients of two m

AzurRx BioPharma has enrolled the first three patients in its Phase 2b trial investigating MS1819 as a treatment for exocrine pancreatic ins

Most autistic people — 87 per cent, according to the latest estimate — have some sort of motor difficulty, ranging from an atypical gait to

Patients with classical-like Ehlers-Danlos syndrome (EDS) due to tenascin-X deficiency, a rare form of EDS, are at risk for tissue fragilit

A Phase 1/2 clinical trial assessing Arrowhead Pharmaceuticals‘ investigational inhaled therapy, ARO-ENaC, for the treatment of cystic fibr

Advocates are calling for rapid government action to protect Victorians living with disability as the number of coronavirus infections lin

Today, the U.S. Food and Drug Administration granted accelerated approval to Viltepso (viltolarsen) injection for the treatment of Duchenne

The financial pressures placed on charities and not for profits during the COVID-19 pandemic are beginning to have an impact on the mental

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to fin