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Surrogate Endpoints: How to Choose the Best One for Your Rare Disease Trial

XTalks

For the following 15 rare diseases, all prevalence (approximate), symptom, etiology and treatment information was taken from the National Organization for Rare Disorders’ (NORD) Rare Disease Database , unless a link to another source is provided. Cushing’s Disease. Acromegaly. Prevalence: 50 to 70 people per 1 million.

Trials 98
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Hypoxia-Inducible Factor-2 Alpha (HIF-2?) Inhibitor WELIREG™ (belzutifan) for the Treatment of Patients With Certain Types of Von Hippel-Lindau (VHL) Disease-Associated Tumors

The Pharma Data

target genes associated with cellular proliferation, angiogenesis and tumor growth. Advise patients of these risks and the need for effective non-hormonal contraception. WELIREG can render some hormonal contraceptives ineffective. Advise patients of these risks and the need for effective non-hormonal contraception.

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Key Trends in the Life Sciences to Look Forward to in 2022

XTalks

RNA therapeutics hold promise as powerful treatments for diseases, including those that are genetic-based. The gene-editing tool CRISPR edits DNA using specialized strands of guide RNA and endogenous cellular mechanisms of RNA interference. RNA Therapeutics. The deal is worth $1.5 Minimally Invasive Devices.