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Home » Topics » Disease categories and therapies » Neonatal/pediatrics

Neonatal/pediatrics
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Illustration highlighting the adrenal glands and kidneys
Cancer

Orphagen's OR-449 receives rare pediatric disease designation for pediatric adrenocortical carcinoma

Jan. 18, 2023
The FDA has awarded rare pediatric disease designation to Orphagen Pharmaceuticals Inc.'s OR-449 for the treatment of pediatric adrenocortical carcinoma (ACC). OR-449 is a selective, first-in-class, potent and orally bioavailable small-molecule antagonist to steroidogenic factor-1 (SF-1; NR5A1), an orphan nuclear receptor and transcription factor that is essential for the growth and development of the adrenal gland.
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Biogen, Eisai win FDA clearance for AD therapy Leqembi

Jan. 10, 2023
By Randy Osborne
As expected, the U.S. FDA gave its go-ahead to lecanemab, an amyloid-beta binder for mild cognitive impairment caused by Alzheimer’s disease (AD) and mild AD from Biogen Inc. and Eisai Co. Ltd, which have assigned to the compound the brand name Leqembi.
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Biogen, Eisai win FDA clearance for AD therapy Leqembi

Jan. 6, 2023
By Randy Osborne
As expected, the U.S. FDA gave its go-ahead to lecanemab, an amyloid-beta binder for mild cognitive impairment caused by Alzheimer’s disease (AD) and mild AD – together known as early AD – from Biogen Inc. and Eisai Co. Ltd, which have assigned to the compound the brand name Leqembi.
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Cancer

Researchers identify anti-CDO Hedgehog inhibitor as strategy to treat rhabdomyosarcoma

Nov. 16, 2022
Rhabdomyosarcoma (RMS) occurs in 3% of all pediatric cancers. Aberrant Hedgehog (Hh) activation can be ligand-dependent or -independent, but current clinical Hh inhibitors targeting SMO are effective only for ligand-independent tumors. Researchers at Vall d’Hebron Institut de Recerca, Barcelona, Spain, developed a targeted therapeutic for Hh ligand-dependent tumors.
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Brain-DNA illustration
Genetic/Congenital

ASP-5736 ameliorates abnormal behaviors in rat model of fragile X syndrome

Oct. 24, 2022
Researchers from Astellas Pharma Inc. presented preclinical data for the novel 5-HT5A receptor antagonist, ASP-5736, being developed for the treatment of fragile X syndrome (FXS).
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Muscular dystrophy
Neurology/Psychiatric

Metriopharm receives grant to support preclinical studies with MP-1032 for DMD

Oct. 13, 2022
Metriopharm AG has received a grant of €125,000 from charity Duchenne UK that will support development of the company's lead compound MP-1032 (luminol sodium salt) for the treatment of Duchenne muscular dystrophy (DMD).
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Cancer

Germline SNP predisposes to low-grade glioma

Oct. 7, 2022
By Mar de Miguel
A germline change in a single nucleotide increased the risk by up to 6-fold of developing an isocitrate dehydrogenase (IDH) mutant low-grade glioma. The rs55705857 genotype could serve as a biomarker before surgery to identify an early glioma.
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DNA illustration
SSIEM 2022

COX11 variants behind infantile-onset mitochondrial encephalopathy

Sep. 5, 2022

Mitochondrial disorders have shown phenotypic and genetic heterogeneity; cytochrome c oxidase assembly protein COX11 is an assembly factor that works as a copper chaperone. Previous findings have unveiled that COX11 knockdown causes a reduction in ATP production in vitro.


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Omnipod 5, Dexcom G6 devices with mobile app on smartphone

FDA clears Insulet’s Omnipod 5 tubeless ‘artificial pancreas’ for children as young as 2

Aug. 26, 2022
By Meg Bryant
Insulet Corp. got the U.S. FDA’s nod to extend use of its Omnipod 5 automated insulin pump to children aged 2 years and up with type 1 diabetes. The expanded indication, just seven months after FDA cleared Omnipod 5 for patients 6 years and older, represents the first time a tubeless automated insulin delivery (AID) system has been authorized for use in children this young.
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Natus logo, teal background overlaying medical concept image

Natus Medical investors sense a sweet payout with Archimed’s acquisition offer of $1.2B

April 18, 2022
By Annette Boyle
Health care-focused private equity firm Archimed Group agreed to acquire Natus Medical Inc. for $33.50 per share, a nearly 29% premium over the April 14 closing share price for the provider of medical devices to diagnose and treat neurological and sensory disorders. The deal will take Natus (NASDAQ:NTUS) private after more than 20 years as a publicly traded company.
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