Gene Therapy
FDA/CDC
FDA approves Zolgensma for infantile-onset SMA treatment
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
New brain circuitry found with Parkinson’s disease gene therapy
Imaging has revealed the formation of new brain circuitry in patients with Parkinson’s disease treated with gene therapy.
Conference Coverage
Investigational gene therapy for medically refractory Parkinson’s shows promise
ATLANTA – A randomized, double-blind, placebo surgery–controlled trial of VY-AADC01 is currently recruiting subjects.
Conference Coverage
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...
Literature Review
Spinal Muscular Atrophy Added to Recommended Uniform Screening Panel
Spinal muscular atrophy (SMA) is now among the disorders officially included in the Recommended Uniform Screening Panel (RUSP), which state public...
Conference Coverage
Strategies for Treating Motor Fluctuations in Parkinson’s Disease
MIAMI—Motor fluctuations in Parkinson’s disease can arise from more than one cause, and a clinician needs to consider a range of possibilities....
Latest News
Spinal muscular atrophy added to newborn screening panel recommendations
It is estimated that national screening will avert 33 annual deaths and 48 annual cases of ventilator dependence.
News
FDA Efforts to Advance Development of Gene Therapies
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...
Conference Coverage
Payers part of the drug-pricing problem, says FDA commissioner
WASHINGTON – FDA Commissioner calls for more competition in biologic products.
News
Experts call for international cooperation on human gene editing
As the International Summit on Human Gene Editing comes to a close, organizers call for cooperation to establish norms for acceptable and ethical...