Growth Disorders
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Panelists acknowledge difficulty generating stronger data in ultra-rare disease
Agency appears unswayed by mitochondria-targeting drug for rare cardioskeletal disease
Association was more apparent when additionally adjusting for BMI
Octreotide subcutaneous depot also improved treatment satisfaction, quality of life
The first spinal muscular atrophy prevalence data based on screening came in lower than expected
Traditional trial-and-error method won't be viable in the near future, researcher says
Randomized trial finds no advantage over immediate stop for teens
Study supports hypothesis that placental abnormalities are intertwined with brain development
Similar neurodevelopmental outcomes, but less necrotizing enterocolitis with donor milk
Cases tied to long-discontinued childhood medical procedure
Myelomeningocele, repeated shunt revision are associated with higher risk in observational study
A therapy to help babies with hole in heart is still lacking
Youth on Jornay PM saw significantly higher weight trajectories
Oral retinoid lands indication for fibrodysplasia ossificans progressiva
