Patients and manufacturers continue on a learning curve to improve trial designs and patient experiences.
Zolgensma is the first gene therapy for the treatment of infantile-onset spinal muscular atrophy in children aged less than 2 years.
From the Journals
Imaging has revealed the formation of new brain circuitry in patients with Parkinson’s disease treated with gene therapy.
ATLANTA – A randomized, double-blind, placebo surgery–controlled trial of VY-AADC01 is currently recruiting subjects.
It is estimated that national screening will avert 33 annual deaths and 48 annual cases of ventilator dependence.
WASHINGTON – FDA Commissioner calls for more competition in biologic products.
As the International Summit on Human Gene Editing comes to a close, organizers call for cooperation to establish norms for acceptable and ethical...