FDA Approves First Nonsteroidal Drug for DMD
U.S. Food and Drug Administration
The FDA granted the approval of Duvyzat™ to Italfarmaco S.p.A
neurodevelopmental disorders
FDA Approves First Gene Therapy for Metachromatic Leukodystrophy
U.S. Food and Drug Administration
The FDA granted approval of Lenmeldy™ to Orchard Therapeutics
First Two Children Receive New Gene Therapy for Rett Syndrome
Neurogene Inc.
Phase 1/2 trial showed the safety and therapeutic potential of administering a novel transgene
Alzheimer’s Progression in Down Syndrome Appears Similar to Other Onset Forms
National Institutes of Health
NIH-funded study suggests people with Down syndrome may benefit from Alzheimer’s disease treatments
Largest Autism Whole Genome Sequencing Study Reveals 134 Autism-Linked Genes
The Hospital for Sick Children
Study sequences the entire genomes of more than 11,000 individuals, offering new insights
Developmental-Behavioral Pediatricians Can Diagnose Most Autism Cases
Boston Children's Hospital
The Autism Diagnostic Observation Schedule did not change the diagnosis in 90 percent of cases
The Use of Telemedicine in Child Neurology
Children's Hospital of Philadelphia
Certain patients were more likely to use telemedicine even with the reopening of in-person appointments
Children with Genetic Disorders More Likely to Also Have Mental Health Problems
University of Cambridge
Researchers hope that this work helps improve the targeting of assessments and interventions
Researchers Use MRI to Show Brain Changes, Differences in Children with ADHD
University of North Carolina Health Care
Researchers documented significant decrease in neural flexibility across the entire brain of children with ADHD
New Genetic Disease That Delays Brain Development in Children
University of Portsmouth
The condition, so new it doesn't have a name yet, causes learning difficulties in patients