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News > Research
The FDA Just Gave a Rare Pediatric Gene Therapy Its Fastest Regulatory Track. Here's What Families of Children with NGLY1 Deficiency Need to Know.
...granted Regenerative Medicine Advanced Therapy (RMAT) designation to GS-100 on April...April 14, 2026, an investigational gene therapy developed by Grace Science...14, 2026, an investigational gene therapy developed by Grace Science for NGLY1...and review of regenerative medicine therapies that show preliminary clinical evidence...
Research > Genetics
CRISPR Gene Therapy for Rare Pediatric Disorders: What Families Need to Know
...the first personalized CRISPR therapy to an infant with CPS1 deficiency,...qualify for personalized CRISPR therapy in the next few years. But...It Doesn't Fix. CRISPR is a gene editing tool. It cuts DNA at...a corrected one, or turns a gene on or off. When it works, it...single mutation in a known gene. If you can fix that mutation,...
Special Needs > Muscular Dystrophy
Muscular Dystrophy in 2026: What Families Need to Know About Gene Therapy and Daily Life
...hearing about gene therapy. Maybe...about gene therapy. Maybe your...FDA-approved gene therapy for...FDA-approved gene therapy for Duchenne...And while gene therapy is...while gene therapy is making headlines,...to evaluate gene therapy as...evaluate gene therapy as an option,...a one-time gene therapy approved...one-time gene therapy approved for...dystrophin gene (the protein...
Special Needs > Cystic Fibrosis
Exploring the Advancements in Cystic Fibrosis Treatments
...caused by a change in the CFTR gene. This gene controls the flow...change in the CFTR gene. This gene controls the flow of salt and...out of cells. When the CFTR gene is not working right, the body...look for changes in the CFTR gene. This is done with a blood test...These include: Chest physical therapy to loosen mucus in the lungs...
Research > Drug Development
Unveiling Breakthroughs in Muscular Dystrophy Treatment
...treatment. Patient safety is the top priority. Promising Research Areas. Gene therapy is one exciting area of research for muscular dystrophy. The goal...treatment. Patient safety is the top priority. Promising Research Areas. Gene therapy is one exciting area of research for muscular dystrophy. The goal is to...
News > Research
FDA Approves First-Ever Gene Therapy for Genetic Hearing Loss, Available for Free
...parvec-cwha), the first gene therapy for...first gene therapy for genetic...of the OTOF gene. Regeneron...FDA-approved gene therapy to...FDA-approved gene therapy to restore...Cause. The OTOF gene produces otoferlin,...copies of the gene are defective...of the OTOF gene directly into...injects the therapy into the cochlea...per ear. The gene therapy restores...ear. The gene therapy restores otoferlin...
News > Research
The Scientists Behind the First Gene Therapies for Blindness and Sickle Cell Just Won $3 Million Prizes. Here's What Families Need to Know.
...Luxturna, the first gene therapy for an inherited...Luxturna, the first gene therapy for an inherited form...first FDA-approved gene replacement therapy...FDA-approved gene replacement therapy for Leber congenital...in infancy. Their therapy, Luxturna, uses a virus...copy of the RPE65 gene directly into eye cells....replaces the defective gene. Penn Medicine reports...
News > Research
New Research Links Immune System 'Brakes' to Autism Behaviors and Points to Possible Treatment
...typically developing children. In an animal model, Treg therapy reduced neuroinflammation, changed gene expression...Treg therapy reduced neuroinflammation, changed gene expression in brain regions tied to autism, and...gut, fewer inflammatory cytokines, and altered gene expression in the cerebellum, frontal cortex, and...
Medical > Cardiology
A Comprehensive Review on Congenital Heart Defects and the Critical Care Demanded in Special Needs Individuals
...procedures that repair defects without necessitating open-heart surgery. Gene editing techniques, still in experimental stages, hold immense potential...especially those with special needs, necessitates a holistic, tailored approach. Therapies beyond medical intervention...
News > Advocacy
Trump Just Signed 100% Tariffs on Brand-Name Drugs. What Disability Families Need to Know About Their Medications.
...200,000 people) Nuclear medicines Plasma-derived therapies Fertility treatments Cell and gene therapies...Plasma-derived therapies Fertility treatments Cell and gene therapies Antibody drug conjugates Medical countermeasures...Plasma-derived therapies Fertility treatments Cell and gene therapies Antibody drug conjugates Medical countermeasures...
News > Research
Scientists Found the First Gene Linked Only to Autism's Social and Repetitive Behaviors. Here's What Families Need to Know.
...identifying PTCHD1-AS, a non-coding RNA gene on the X chromosome, as specifically...is the first time a non-coding RNA gene has been definitively tied to autism's...the findings could lead to precision therapeutics for all autism diagnoses. What...mouse models engineered to lack this gene, male mice showed increased repetitive...
Research > Genetics
Chromosomal Microarray vs. Whole Genome Sequencing: Which Genetic Test Is Right for Your Child?
...disease. If your child has a rare genetic condition caused by one broken gene, WES is more likely to catch it than CMA. Whole genome sequencing (WGS) reads...(VUS), meaning results your doctors can't interpret yet. CMA won't find single-gene mutations. WES won't...