Results
Lifestyle > Independence
How to Organize the Chaos
...acquire, understand, utilize and store pertinent medical information and knowledge makes us better informed and more actively involved with our own medical...cancer a mere three hours after my daughter was diagnosed with Crohn's disease. We were simultaneously thrown into a world of second opinions, diagnostic...
Parenting > Adulthood
Successful Transitions: Focusing on Medical Tasks
...responsibility of medical tasks. Knowledge of Disease, Medical Requirements,...of medical tasks. Knowledge of Disease, Medical Requirements, and Medical...to change. In addition to medical knowledge, there are also the financial, insurance,...help you know what types of medical knowledge and tasks should be taught at the...
Special Needs > Cancer
The Role of Technology in Enhancing Cancer Care for Special Needs Patients
...we have gained an unprecedented understanding of our susceptibility to diseases and potential responses to therapies. This invaluable information empowers...that influence our risk of developing certain conditions. Armed with this knowledge,...
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.
...families of children with this ultra-rare genetic disease, this marks the first treatment specifically targeting...around 12 children are born with it each year. The disease affects multiple body systems and is characterized...evidence of addressing serious or life-threatening diseases. For families, this means three things. First,...
Research > Genetics
CRISPR Gene Therapy for Rare Pediatric Disorders: What Families Need to Know
...turns a gene on or off. When it works, it addresses the genetic cause of a disease rather than managing symptoms. That precision is what makes it powerful...mutation in a known gene. If you can fix that mutation, you can stop the disease progression. But CRISPR doesn't work for every rare disorder. It's most...
Research > Genetics
Chromosomal Microarray vs. Whole Genome Sequencing: Which Genetic Test Is Right for Your Child?
...protein-coding portions of your child's genome, about 1-2% of the total. It's good at finding point mutations (single-letter changes in DNA) that cause 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...
Research > Funding
Private Foundations Funding Disability Research: A Guide for Advocates and Families
...This lag reflects institutional blind spots, not active avoidance. Small patient populations don't 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...
Special Needs > Rare Disorders
Ending the Diagnostic Odyssey: How Whole Genome Sequencing Is Changing Rare Disease Diagnosis
The average rare disease diagnosis takes 5 to 7 years and visits to 8 different...dealing with. This pattern has a name in the rare disease community: the diagnostic odyssey. In 2026, whole...your genome doesn't code for proteins, and some disease-causing variants hide in those regulatory regions....
Special Needs > Respiratory Health
Respiratory Health for Children with Disabilities: A Complete Family Guide
Covering asthma, chronic lung disease, and ventilator dependence with practical management strategies and advocacy guidance.
Special Needs > Multiple Sclerosis
Living with Pediatric MS: A Complete Guide for Families
...You're stepping into a world of neurologists, MRIs, disease-modifying therapies, and school meetings where...Pediatric MS. Multiple sclerosis is an autoimmune disease where the immune system attacks myelin, the protective...rate early on is real. It's also why starting disease-modifying therapy early matters. Recognizing Symptoms....
Special Needs > Down Syndrome
Heart Defects in Down Syndrome: What Every Parent Should Know
...mean for your child's long-term health. The Most Common Heart Defects in Down Syndrome. Three defects account for the majority of congenital heart disease in children with Down syndrome: atrioventricular septal defect (AVSD), ventricular septal defect (VSD), and atrial septal defect (ASD). Atrioventricular...
Research > Drug Development
The Drug Development Pipeline for Special Needs: What Parents Need to Know About How Treatments Go from Lab to FDA Approval
...be waiting. The path from laboratory discovery to FDA approval spans 12 to 15 years on average. That timeline holds across conditions, though rare disease drugs can move faster under specific circumstances. The process isn't arbitrary. Each stage answers a specific question about whether a drug is safe...