Genetic Disease, Hormones and Licensing - Clinical Research Informer

Neurocrine Biosciences’ Crenessity Approved as First New Treatment in Decades for Rare Genetic Disorder CAH

XTalks

DECEMBER 18, 2024

CAH is a group of rare genetic disorders affecting the adrenal glands, which produce essential hormones like cortisol, aldosterone and androgens. The most common form of CAH, 21-hydroxylase deficiency, disrupts hormone production, leading to cortisol deficiency and an overproduction of androgens. billion in revenue by 2024.

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

XTalks

SEPTEMBER 17, 2020

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. What Is a Surrogate Endpoint? Acromegaly.

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Clinical Research Informer

Neurocrine Biosciences’ Crenessity Approved as First New Treatment in Decades for Rare Genetic Disorder CAH

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

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