A new UCLA Health study has discovered in mouse models that genes associated with repairing mismatched DNA are critical in eliciting damages to neurons that are most vulnerable in Huntington's disease ...

Huntington's disease is a neurodegenerative disorder that is usually fatal about 15 to 20 years after a patient is diagnosed. It is known ... | Genetics And Genomics ...

Treetops Hospice was filled with a colourful display of mismatched socks this week, as the Huntington’s Disease peer support ...

Under the terms of the agreement, Novartis will pay $925 million upfront and up to $2.15 billion, contingent upon achievement ...

"The Evrysdi tablet combines established efficacy with convenience, providing an additional flexible option for SMA management." The approval of the Evrysdi tablet was based on the results of a ...

Evrysdi is the only non-invasive disease-modifying SMA treatment and is approved in over 100 countries Evrysdi tablet can be stored at room temperature and offers the same demonstrated efficacy and sa ...

The acting chair of the, Mark Uyeda, took the first step Tuesday to rolling back a rule that would require thousands of ...

One in 10 Americans live with a rare disease, which often lacks effective treatments. Such is the case for PIGA-CDG, an ultra ...

In a new JAMA Dermatology study, scientists at King's College London found that changes in two parts of the genome work ...

Over 1,000 genes may serve as possible treatment targets for individuals with kidney disease, according to a new study. By creating the most complete and detailed genetic 'map' of kidney function to ...

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has approved a New Drug Application (NDA) for an Evrysdi® ...