There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University's College ...

Researchers hoped they had finally found a way to get gene therapies past the blood-brain barrier. Then the first patient ...

The FDA has issued its stamp of approval to a new, cell-based option to treat Wiskott-Aldrich syndrome (WAS), marking the ...

Cure Rare Disease (CRD) announced it has been awarded a $7.4 million grant from the California Institute for Regenerative ...

The treatment uses a modified, harmless virus to deliver healthy genes to a patient’s cells to treat genetic diseases. Read ...

Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a ...

A new class of life-saving drugs is helping children who once had no hope. But some carry a price tag of millions for a ...

Qalsody is one step closer to being covered by public Canadian healthcare systems when used to treat adults with ALS with ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University’s College ...

On a video call in early September, Sarah Tabrizi first saw the data that she and other researchers studying Huntington’s ...

Using X-ray diffraction data, James Watson (1928-2025) made the seminal discovery of the double-helical structure of DNA ...

Caden Major was born with sickle cell disease – an inherited blood disorder affecting red blood cells. Now at 19 years old, ...