Drugs, Gene Silencing, Genetic Disease and Genetics

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Drugs

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Genetic Disease

Genetics

UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD

XTalks

FEBRUARY 14, 2022

The UK’s National Health Service (NHS) has recommended the use of Libmeldy for the treatment of the rare genetic disease metachromatic leukodystrophy (MLD). After further discussion with Orchard Therapeutics, the NHS negotiated an additional, undisclosed discount on the drug, allowing the health agency to now offer it to patients.

Gene Therapy

Gene Therapy Drugs Gene Gene Silencing

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

The Pharma Data

NOVEMBER 24, 2020

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. PH1 is an ultra-rare genetic disease characterized by oxalate overproduction.

FDA Approval

FDA Approval Production RNA Medicine

Clinical Research Informer

UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD

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

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