Following up on previous, dimly received issuances, a new set of ideas published by the FDA to streamline regulatory pathways ...

The neurodegenerative disease damages patients' motor, cognitive and behavioral controls, which sometimes manifest as ...

Breakthrough Therapy designation granted based on FDA’s review of available clinical evidence of safety and efficacy from all ...

Novo Nordisk has a factor VIII mimetic in a phase 3 trial, and a Chinese company is forging ahead with its gene therapy ...

Treatment introduces an expansion of the scope of gene therapies that might lead to similar treatments for neurodegenerative ...

A gene therapy trial claims to show promising results for patients with Huntington’s disease. A press announcement of the experimental treatment suggests significant slowing of the disease over three ...

University College London's National Amyloidosis Center leads a multinational team reporting that a single infusion of an in ...

How cell and gene therapy developers can lessen the chances of receiving FDA complete response letters through the use of ...

Managed Healthcare Executive gives C-suite executives in health plans and provider organizations news and strategies for ...

Rocket has pulled an approval application to the FDA, with the biotech considering offloading the asset via external ...

The MCO-010 vector uses surviving cells in the retina to produce a photosensitive protein that may help patients regain some ...

The company said that the decision is based on business and strategic considerations and does not reflect concerns regarding ...