Please provide your email address to receive an email when new articles are posted on . Device-based therapy may be necessary for certain patients with NYHA class II/III heart failure with reduced ...

An ultra-rare enzyme deficiency that keeps infants from achieving proper muscle function among other developmental delays now has its first FDA-approved treatment. The new product, a gene therapy ...

The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. Now, seven years and many approvals later, innovation in the space has hit new heights with PTC ...

PTC Therapeutics announced this week that it is halting its preclinical and early research in gene therapy as part of “a strategic portfolio prioritization.” Instead, the biopharma will pour most of ...

PTC Therapeutics’ Upstaza, a gene therapy for an ultra-rare genetic disorder and the first to be directly infused in the brain, is one step closer to reaching patients in the U.K. The National ...