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.
Gene-Replacement Therapy for SMA1 May Necessitate New Rating Measures to Capture Patients’ Motor Function Gains
CHICAGO—The Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) was designed to track motor function...
Spinal muscular atrophy (SMA) is now among the disorders officially included in the Recommended Uniform Screening Panel (RUSP), which state public...
MIAMI—Motor fluctuations in Parkinson’s disease can arise from more than one cause, and a clinician needs to consider a range of possibilities....
It is estimated that national screening will avert 33 annual deaths and 48 annual cases of ventilator dependence.
Once just a theory, gene therapies are now a therapeutic reality for some patients. These platforms may have the potential to treat and cure some...
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...