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No adverse events resulted in treatment discontinuation

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