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Clinical Trials

Gene Silencing

Genetic Disease

Medicine

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). Related: Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data. million (approximately $3.8 million USD).

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

PH1 is an ultra-rare genetic disease characterized by oxalate overproduction. We are grateful to all the investigators, staff and patients who participated in the ILLUMINATE clinical studies, and to their families, caregivers and patient advocates. President of R&D at Alnylam.

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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