BOSTON, Mass. — Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, announced today that the U.S. Food and Drug Administration (FDA) has approved JUXTAPID® (lomitapide) capsules for pediatric use in homozygous familial hypercholesterolemia (HoFH) for...
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BOSTON, Mass. — Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases, is pleased to announce the Health Canada approval of MYALEPTA™ (metreleptin for injection). As an adjunct to diet, MYALEPTA is indicated as a...
BOSTON, Mass. — Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, today announced the publication of results from a Fabry disease patient survey in the peer-reviewed Orphanet Journal of Rare Diseases. “There continues to...
BOSTON, Mass. — Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases, announced today that the U.S. Food and Drug Administration (FDA) approved FILSUVEZ® (birch triterpenes) topical gel for the treatment of partial thickness wounds...
CARLTON, Mercer County — Kyle Bennett is a 4-year-old, and he looks and behaves as such. It’s all shy smiles at first, then he opens up to show you what he’s into — kid stuff like his puppy, fishing with Dad, games, Scooby Doo … and more Scooby Doo. Kyle...
COPENHAGEN, Denmark — Ascendis Pharma A/S (Nasdaq: ASND) today announced new radiographic data from Week 104 from the completed pivotal ApproaCH Trial of once-weekly TransCon CNP (navepegritide) in children with achondroplasia. In the trial, TransCon CNP-treated children demonstrated continued improvements in lower extremity alignment through up to two years of...
Denver, Colorado – Four children who acquired HIV in utero have remained free of detectable HIV for more than one year, according to new findings that were presented on March 6, at the Conference on Retroviruses and Opportunistic Infections (CROI) in Denver, Colo. The study is part of ongoing, multinational...
PHILADELPHIA, PA — Researchers at Children’s Hospital of Philadelphia (CHOP) developed a longitudinal atlas of neuroblastoma, a common and potentially deadly childhood cancer, to gain deeper understanding into precise molecular mechanisms underlying why and how certain treatments eventually become ineffective. The findings, which offer insights that could potentially lead to new personalized medicine approaches...
Philadelphia, PA – In a landmark study, an international consortium led by researchers at Children’s Hospital of Philadelphia (CHOP) published the final results of a key clinical trial of the gene therapy CASGEVY (exagamglogene autotemcel) for the treatment of sickle cell disease in patients 12 years and older with recurrent...
Washington, DC — Children’s National Hospital and the Gilbert Family Foundation today announced a new $8 million, five-year grant to support groundbreaking research on neurofibromatosis type 1 (NF1). The grant, which is part of Gilbert Family Foundation’s Brain Tumor Initiative (BTI), will accelerate investigations aimed at improving the lives of...