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Hope wins over Fear, America may be "First" but a divided nation creates an unstable world. Congratulations President-Elect @JoeBiden

Tracy Murray has witnessed a lot of change in her 27 years of work in classrooms. But in her view, no shift has been as radical—or as posit

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 EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pu

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

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

The human capacity to embrace change and pivot rapidly has been seen as one of the few positives to emerge out of the gloom of the pandemic.

Illustration of people of diverse racial backgrounds. With the anniversary of the Americans With Disabilities Act, a lot of disabled peop

Inherited mutations in a gene that keeps nerve cells intact were shown, for the first time, to be a driver of neuropathy known as Charcot-Ma

Researchers have developed the first thread-based flexible patch that can be sewn into clothing to monitor — in real-time — several molecule

Orion Corporation’s oral therapy levosimendan (ODM-109) failed to preserve lung function and overall functionality in people with amyotrophi

New discoveries about the disruption of condensates in the neurodevelopmental disorder Rett syndrome provide insights into how cells compart

This is the story of a fatal genetic disease, a tenacious scientist and a family that never lost hope.

A new mutation in the FUS gene, which results in a shorter version of the FUS protein that cannot enter form the cell nucleus to work as it

Superfest, the world’s longest disability film festival, continued its ADA-30 celebration with Disability Pride Philadelphia by screening fi

Today, we reflect on the progress we have made as a Nation in securing equal rights and defending the inherent dignity of all Americans.

Mutations in the MECP2 gene, which lead to most cases of Rett syndrome, prevent the formation of droplet-like structures that regulate DNA.