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The therapy is approved to be used in combination with glucocorticoid replacement therapies (steroids) to control androgen levels in adults and pediatric patients four years of age and older with classic CAH. Currently, the standard treatment for CAH involves glucocorticoids, which replace cortisol and help regulate hormone levels.
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.
This included Eli Lilly’s partnership with Netherlands-based RNA biotech ProQR Therapeutics this year to develop RNA editing-based therapies for rare geneticdiseases, namely blindness-causing retinal conditions, based on the company’s proprietary Axiomer RNA editing platform technology. The deal is worth $1.5
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