RESTORE is one of the largest long-term follow-up studies for a rare disease treatment, with 62 participants accepting the invitation to enroll. All participants were treated with a single ...
The "Transfection Reagents & Equipment market by Product, by Method, by Application, by End-User, and By Region" report has been added to ResearchAndMarkets.com's offering. The global transfection ...
Amid recent backlash stemming from market withdrawals and trial delays, the FDA seeks to further clarify its requirements for confirmatory trials for therapies seeking the shortened path to market.
Five years after the one-time injection, patients with Leber Hereditary Optic Neuropathy (LHON) due to the MT-ND4 gene variant demonstrated sustained bilateral improvement in visual acuity and favorab ...
Pfizer gave Sangamo Therapeutics coal for Christmas, terminating a hemophilia A gene therapy pact to deprive the cash-strapped biotech of up to $220 million in milestones. Pfizer’s decision sent ...