Results
Research > Genetics
CRISPR Gene Therapy for Rare Pediatric Disorders: What Families Need to Know
...mutation, built from scratch as a one-time intervention outside any clinical trial. For families navigating rare pediatric diagnoses, that news raises the...infrastructure being built right now, including the research centers, clinical trials, and specialized expertise at major children's hospitals, will determine...
Research > Funding
Private Foundations Funding Disability Research: A Guide for Advocates and Families
...generate the donor urgency that cancer or heart disease do. Academic prestige doesn't cluster around disability research the way it does around oncology trials. The result is a funding ecosystem where federal support dominates, and when federal budgets contract, entire research areas stall. Who's Funding What...
Research > Drug Development
Should Your Child Participate in a Clinical Trial? A Parent's Decision Framework
...specialist just mentioned a clinical trial. Or an advocacy organization sent an...structured way to evaluate whether this trial makes sense for your family. Here's...through that decision. What Phase Is the Trial, and What Does That Mean? Trials move...the Trial, and What Does That Mean? Trials move through three phases before FDA...
Research > Drug Development
Fragile X Drug Trials: The First Real Breakthroughs and What They Mean for Families
...and all three failed in human trials between 2009 and 2015. Families...skepticism into the way they read trial announcements. Which is exactly...positive results in a Phase 2 trial for Fragile X syndrome. Not...that the FDA greenlit Phase 3 trials, the final confirmatory step...it means. Why Those Earlier Trials Failed. The mGluR5 hypothesis...
Research > Assistive Tech
Brain-Computer Interfaces for Children Who Can't Speak: Research, Reality, and How to Access Trials
...now from what's being tested, explains why pediatric BCI design is fundamentally different from adult applications, and shows you exactly how to find trials your child might qualify for. What Brain-Computer Interfaces Are. A brain-computer interface measures brain activity and translates it into commands...
News > Research
What Evidence-Based Really Means in Special Education and Therapy
...across thousands of participants. A therapy backed by one small pilot study can use the same label as a treatment tested in 40 randomized controlled trials. Both are technically "evidence-based." The quality of that evidence, though, is worlds apart. When you're evaluating a therapy recommendation, choosing...
Therapies > Other
Alternative and Complementary Therapies for Special Needs Children: What the Research Says
...carries equal weight. When evaluating whether a therapy works, the quality of evidence matters as much as whether studies exist. Randomized controlled trials (RCTs) compare a therapy group to a control group. Participants are randomly assigned. This design isolates the therapy's effect from placebo, natural...
Research > Genetics
Understanding Genetic Testing for Autism, Cerebral Palsy, and Developmental Disabilities: A Parent's Guide
...genetic cause that could affect treatment decisions, connect you to clinical trials, or inform family planning. For some families, it provides answers. For...existed. A specific genetic diagnosis may qualify your child for clinical trials testing new therapies. It can guide medical monitoring for related health...
Research > Drug Development
The Drug Development Pipeline for Special Needs: What Parents Need to Know About How Treatments Go from Lab to FDA Approval
You see a headline about a promising autism drug entering Phase 2 trials, and you wonder if this could be the treatment your child needs. But Phase...you're making decisions about your child's care, evaluating whether to join a trial, or trying to gauge how long you might be waiting. The path from laboratory...
Research > Clinical Trials
Why Children with Disabilities Are Underrepresented in Clinical Trials
...ineligible for the trial that could help them....this way. Clinical trials systematically exclude...appears frequently in trial exclusion lists. It...is: we designed this trial for the easiest version...not edge cases. But trials exclude them because...Phase 1 and Phase 2 trials exclude medically complex...participants, Phase 3 trials follow the same pattern....
Research > Clinical Trials
The Questions Every Parent Should Ask Before Enrolling in a Clinical Trial
You've found a trial. The eligibility...designed to filter out trials. They're the framework...specific risks of this trial compared to standard...face without the trial. If the trial involves...the trial. If the trial involves a new medication,...If it's a device trial, ask about failure...invasive are the trial procedures compared...already doing? Some trials require only what...
Research > Clinical Trials
How to Find and Evaluate Clinical Trials for Your Child's Disability
...heard about a clinical trial that might help your child....central registry. Every trial in the U.S. that receives...the database, what the trial phases mean for your child,...for evaluating whether a trial is the right fit. How..."Not yet recruiting." Trials marked "Completed" or...child's age range. Pediatric trials often have narrow age...