A 'fit and healthy' woman was left paralysed after headaches she thought were 'work stress' turned out to be signs of an ...

Precision BioSciences Inc., a clinical-stage company developing gene editing therapies for diseases with high unmet need, has ...

The deal with Arrowhead's Visirna Therapeutics subsidiary – which also includes $265 million in milestones tied to potential ...

The accountant began suffering from severe headaches during her 20s until she was diagnosed with brain arteriovenous ...

Europe risks ceding its leadership in rare disease innovation unless it urgently recalibrates its incentive framework, warns Enrico Piccinini, Senior Vice President EU & International at Chiesi.

Biogen’s Qalsody (tofersen) has been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) as the first ...

A Senate committee has rejected President Donald Trump’s efforts to gouge funding for the National Institutes of Health (NIH) ...

A genetic technology can treat Hutchinson-Gilford progeria syndrome, a rare and incurable disease that causes rapid aging ...

Pegcetacoplan inhibits C3 activation by binding to C3 and its activation fragment C3b, thereby reducing C3 glomerular fragment deposition in patients with C3G and primary IC-MPGN.

Treatment options for patients with epithelioid hemangioendothelioma vary dependent on disease status, but more therapies are ...

The parents of a five-year-old boy say they "constantly worry every day" because he has a rare condition, which means "there ...