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Regulating therapies for rare diseases – recent approvals
This article summarises some of the recent notable drug approvals in the EU and US, including a gene therapy and an enzyme replacement therapy.
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IgA nephropathy
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Novartis releases new data for innovative rare kidney disease treatment
Data from the pre-specified interim analysis of Fabhalta® (iptacopan) indicate a statistically significant reduction in proteinuria reduction.
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Developing a new treatment paradigm for IgAN
On World Kidney Day 2024, Dr Marshall Fordyce, CEO of Vera Therapeutics explores the challenges of treating kidney disease and shares why the company’s lead candidate could become the first self-administered B-cell modulation therapy.
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CHMP meeting highlights: February 2024
Ten new medicines, including a new oligonucleotide therapy for ALS, were recommended for approval at the Committee for Medicinal Products for Human Use (CHMP)’s recent meeting.
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£2.5b acquisition to boost Novartis’ renal portfolio
As part of its planned £2.5 billion acquisition of Chinook Therapeutics, Novartis will gain access to a targeted biologic for a rare kidney disease.
