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US FDA approves Vertex’s Orkambi for cystic fibrosis in children

Pharmaceutical Technology

The US Food and Drug Administration (FDA) has approved the expanded usage of Vertex Pharmaceuticals ’ Orkambi (lumacaftor/ivacaftor) to treat cystic fibrosis (CF) in children aged 12 months to under 24 months. It enrolled 46 children aged one to under two years with the F/F genotype.

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FDA grants approval for bluebird’s Zynteglo to treat beta-thalassemia

Pharmaceutical Technology

The US Food and Drug Administration (FDA) has granted approval for bluebird bio ’s Zynteglo (betibeglogene autotemcel, beti-cel) for the treatment of the underlying genetic cause of beta?thalassemia 0 genotypes. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Top 30 New Medical Devices of 2024

XTalks

TriClip G4 System Manufacturer/developer : Abbott Medical Date of FDA approval : April 1, 2024 Approved for : Tricuspid regurgitation (TR). Date of FDA approval : March 29, 2024 Approved for : To detect exposure to human parvovirus B19. TriClip size comparison photo. Photo courtesy of Abbott Medical.

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FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1

The Pharma Data

FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1. Food and Drug Administration (FDA) approved Oxlumo (lumasiran) injection for subcutaneous use, the first-ever therapy available for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.

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BridgeBio Pharma’s Nulibry Approved as First Treatment for Molybdenum Cofactor Deficiency Type A

XTalks

Today’s action marks the first FDA approval for a therapy to treat this devastating disease,” said Hylton V. Joffe, MD, director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research, in a press announcement from the FDA. “The

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Navigating Neuroscience Trials: Biomarkers, Imaging and CRO Strategies

XTalks

Additionally, the potential for accelerated approval of therapies via biomarker surrogate endpoints is introducing new complexities into the mix. “In This approval was based on the observed reduction of plasma neurofilament light chain (NfL) in patients who received tofersen.

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Rare Kidney Diseases: Exploring The Uncommon Rare Conditions

Roots Analysis

Absence of Biomarkers for Diagnosis: Due to the absence of genotype-phenotype correlations and prognostic markers, there have been challenges in the diagnosis and treatment of rare kidney diseases. Insufficient Model Organisms: Limitations of these model organisms includelong generation time and strain effects.