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Pediatric Rare Diseases New

Written by Dr. Shmuel Prints and Yana Prints.

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4/17/2019 | NIH

Anti-inflammatory drug halts strokes in children with rare disease

4/17/2019 | NIH

Gene therapy restores immunity in infants with rare immunodeficiency disease

12/4/2018 | MD Magazine

Michael Jordan, MD, Differentiates Emapalumab Treatment in Pediatric Patients Versus Adults with HLH

Updates

7/12/2019 | AJMC

Tezacaftor, Ivacaftor Combination Generally Safe, Well Tolerated in Children With CF Under Age 12

Jun 19 2019 | News Medical Life Sciences

Researchers identify genes linked with survival in children with biliary atresia

Jun 19 2019 | News Medical Life Sciences

Scientists pinpoint cause of fatal respiratory disorder that turns babies' lips and skin blue

5/24/2019 | FDA

FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality

5/9/2019 | AJMC

FDA Approves First Treatment for Pediatric Lambert-Eaton Myasthenic Syndrome

5/7/2019 | News Medical Life Sciences

Bioactive compounds in cocoa bean extract could be used to develop new treatment for skeletal diseases

Jun 20, 2018 | The Scientist

Positive Trial Results for Experimental DMD Gene Therapy