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Growth Disorders

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Elamipretide for Barth Syndrome Survives FDA Advisory Vote

Panelists acknowledge difficulty generating stronger data in ultra-rare disease

Oct 11, 2024
FDA ADCOMM elamipretide hydrochloride injection over a computer rendering of mitochondria.
FDA Reviewers Cast Doubt on Drug Candidate for Barth Syndrome

Agency appears unswayed by mitochondria-targeting drug for rare cardioskeletal disease

Oct 09, 2024
FDA ADCOMM elamipretide hydrochloride injection over a computer rendering of mitochondria.
Maternal PCOS Linked to Growth Restriction in Newborns

Association was more apparent when additionally adjusting for BMI

Aug 27, 2024
A computer rendering of polycystic ovary syndrome.
Investigational Once-Monthly Injectable Boosts Response Rate in Acromegaly

Octreotide subcutaneous depot also improved treatment satisfaction, quality of life

Jul 23, 2024
A computer rendering of the brain with the pituitary gland highlighted.
Newborn Screening Downgrades Prevalence of SMA

The first spinal muscular atrophy prevalence data based on screening came in lower than expected

Jul 15, 2024
 A photo of a newborn undergoing a heel prick screening test.
Faster Hormonal Control With Personalized Acromegaly Treatment Approach

Traditional trial-and-error method won't be viable in the near future, researcher says

Jul 09, 2024
A photo of the hands of a man affected by acromegaly next to a normal female hand.
No Need to Wean Off of Scoliosis Bracing

Randomized trial finds no advantage over immediate stop for teens

Jun 03, 2024
A photo of a mother helping her teen daughter get her scoliosis brace on at their home.
Placenta Issues Linked to Newborns' Brain Aberrations in Congenital Heart Disease

Study supports hypothesis that placental abnormalities are intertwined with brain development

Mar 01, 2024
A computer rendering of a fetus in the womb
Donor Milk, Formula Go Head-to-Head for Extreme Preemies in the NICU

Similar neurodevelopmental outcomes, but less necrotizing enterocolitis with donor milk

Jan 31, 2024
A photo of a premature infant in an incubator.
Alzheimer's May Be Transmissible in Rare Circumstances

Cases tied to long-discontinued childhood medical procedure

Jan 29, 2024
A computer rendered progression of neuron damage in Alzheimer’s disease
Shunted Infantile Hydrocephalus Linked to Early Puberty in Girls

Myelomeningocele, repeated shunt revision are associated with higher risk in observational study

Jan 29, 2024
A computer rendering of a baby with a transparent brain with enlarged ventricles highlighted
Ibuprofen Fails Another Crack at Reducing Preemie Lung Issues and Deaths

A therapy to help babies with hole in heart is still lacking

Jan 24, 2024
A photo of a woman holding a premature infant
Delayed-Release ADHD Therapy Slightly Mitigates Slowed Growth in Kids

Youth on Jornay PM saw significantly higher weight trajectories

Sep 09, 2023
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First Therapy Approved for Ultra-Rare Bone Disorder

Oral retinoid lands indication for fibrodysplasia ossificans progressiva

Aug 16, 2023
FDA APPROVED palovarotene (Sohonos) over a photo of a skeleton of a fibrodysplasia ossificans progressiva sufferer.
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