Early data are due for Krystal Biotech’s epidermolysis bullosa project and Uniqure’s haemophilia B candidate.
As data on Amicus Therapeutics' clinical-stage Pompe disease candidate roll in, the group locks up preclinical gene therapy assets.
The $6.6bn of M&A deals announced by drug makers in third quarter of 2018 marks the slowest three-month period since 2013.
Companies that want to play in rare diseases need to shop for gene therapies, but price tags are looking rich.
The FDA asks Amicus for more data on its Pompe disease candidate, but the company is already looking towards a Fabry disease gene therapy, and has made a remarkable…
Bristol-Myers Squibb should soon unveil data showing whether Opdivo’s liver cancer use could be broadened from second to first line, while Amicus investors look for a…
A looming interim analysis could make Amryt a player in epidermolysis bullosa – and boost its deal-making ambitions.
The Bristol-Myers Squibb and Amicus drugs face approval decisions next week.
Will US right-to-try legislation be a means of giving hope to terminally ill patients, or drug another way for drug regulatory standards to fall?