FDA

News

Xenpozyme is first FDA-approved drug for rare disease ASMD

Sanofi now has approval on both sides of the Atlantic for Xenpozyme – its enzyme replacement therapy for rare disease acid sphingomyelinase deficiency (ASMD) – after getting a green light f

R&D

Automation in the clinical laboratory. Pipetting robot laboratory. Medicine robotics. Research and science background

Expectations rise for gene therapy to change treatment lands...

After a recent approval, there are now three gene therapies available on the US market.

Digital

Health Innovators – Amy Abernethy and Jennifer Goldsack Part...

Verily’s Amy Abernethy and DiMe’s Jennifer Goldsack return for a second conversation on data and healthcare, this one focusing on specific use cases and examples.

Patients

Colorful painted group of people figures

To build diversity into clinical trials, collaborate at the ...

Diversity in clinical trials is a “scientific imperative”, but how can industry bridge the gap between “why” and “how”?

News

bluebird unveils $2.8m price for gene therapy Zynteglo on FD...

bluebird bio's Zynteglo has become the first cell-based gene therapy to be approved in the US, getting the nod from the FDA as a treatment for patients with beta thalassaemia who require re

R&D