Rare Disease Treatment Saves Boy After 5-Year Wait
For two years, parents suctioned their son's airway every five minutes to keep him alive. A new treatment changed everything in just weeks.
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Toddler Breathes Free After Rare Disease Treatment at NIH
For two years, parents suctioned their son's airway every five minutes to keep him alive. Then an experimental treatment changed everything.
New Hunter Syndrome Drug Offers Hope to 500 U.S. Families
After 20 years, the FDA approved the first treatment for Hunter syndrome that can prevent brain damage in children. For families watching their kids lose the ability to speak and walk, it's a breakthrough they feared would never come.
FDA Approves Higher Dose SMA Treatment for Faster Results
A new high dose version of an SMA treatment just won FDA approval, offering faster results for babies and children fighting this devastating muscle disease. The breakthrough gives families a more powerful option against a condition that once had no treatment at all.
FDA Approves First Drug to Cross Blood-Brain Barrier
Children with Hunter syndrome now have access to the first treatment that reaches the brain, addressing complications that existing therapies couldn't touch. The FDA approval opens doors for treating dozens of other neurological conditions.
Baby Teeth Stem Cells Show Promise for Cerebral Palsy
Japanese researchers successfully treated cerebral palsy symptoms in rats using stem cells from baby teeth, even after disabilities had already developed. Clinical trials are now underway to test this safe, ethical treatment in children.
3-Year-Old Walks Again After 20 Minutes Underwater
A Vietnamese toddler pronounced likely vegetative after a near-fatal drowning is now walking and talking, thanks to an experimental treatment his doctors provided free of charge. His recovery is rewriting what's possible for children with severe brain injuries.
Gene Therapy Helps Boys Walk Stronger 3 Years After Treatment
Boys with Duchenne muscular dystrophy are showing sustained improvements three years after receiving Elevidys gene therapy, demonstrating the treatment can slow progression of this muscle-wasting disease. The results offer hope to families facing a condition that typically causes rapid physical decline around age 9.