Gene Therapy
Conference Coverage
Genetic therapies bring change to neurology clinics
Advancements are set to alter the approach to some patients and inspire a demand for genetic counselors in neuromuscular clinics.
News
Why genetic testing may be our best shot at progress in Parkinson’s disease
A growing body of work has prompted researchers and drug developers to take a longer look at how genetics influence Parkinson’s disease risk and...
Conference Coverage
Gene therapy demonstrates modest success in genetic blindness
‘First step’ toward improvement in Leber hereditary optic neuropathy is maintained out to 4 years.
Conference Coverage
Survey identifies clinicians’ unease with genetic testing
A survey of Movement Disorders Society members identified a number of gaps in clinicians’ comfort levels with genetic testing for Parkinson’s...
Conference Coverage
Families face challenges of gene therapy
Patients and manufacturers continue on a learning curve to improve trial designs and patient experiences.
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.
Sponsored Supplement
Fundamentals of Gene Therapy: Addressing Gaps in Physician Education
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....