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Patricia Fechner, MD

Can you provide an overview of CAH, how the diagnosis is made with newborn screening, and what the current deficiencies are in therapy?

Can you provide an overview of CAH, how the diagnosis is made with newborn screening, and what the current deficiencies are in therapy?

Patricia Fechner, MD

What is the current mainstay of treatment for CAH? What are the short- and long-term complications of present approaches—based on corticosteroid therapy—that encourage development of alternative therapies?

What is the current mainstay of treatment for CAH? What are the short- and long-term complications of present approaches—based on corticosteroid therapy—that encourage development of alternative therapies?

Patricia Fechner, MD

What are the current dosages for glucocorticoid therapy and what markers are currently followed to assess adequacy of therapy? And why do clinicians have an incentive to lower glucocorticoid dose in persons with CAH?

What are the current dosages for glucocorticoid therapy and what markers are currently followed to assess adequacy of therapy? And why do clinicians have an incentive to lower glucocorticoid dose in persons with CAH?

Patricia Fechner, MD

Does the pediatric endocrinologist have reasons to be concerned about toxicities, side effects, and/or complications associated with long-term corticosteroid use in the pediatric patient with CAH?

Does the pediatric endocrinologist have reasons to be concerned about toxicities, side effects, and/or complications associated with long-term corticosteroid use in the pediatric patient with CAH?

Patricia Fechner, MD

Given the co-morbidities associated with supra-physiologic doses of corticosteroids, what is the rationale for inhibiting the hypothalamic-pituitary-adrenal axis upstream, by using the CRF1 receptor antagonist crinecerfont?

Given the co-morbidities associated with supra-physiologic doses of corticosteroids, what is the rationale for inhibiting the hypothalamic-pituitary-adrenal axis upstream, by using the CRF1 receptor antagonist crinecerfont?

Patricia Fechner, MD

What are the unique elements of the trial design for the CAHtalyst trial, which evaluated crinecerfont in children and adolescents for the treatment of congenital adrenal hyperplasia?

What are the unique elements of the trial design for the CAHtalyst trial, which evaluated crinecerfont in children and adolescents for the treatment of congenital adrenal hyperplasia?

Patricia Fechner, MD

What were the findings from the CAHtalyst trial in terms of the ability of the CRF1 receptor antagonist crinecerfont to reduce hydrocortisone dose while maintaining acceptable androstenedione levels?

What were the findings from the CAHtalyst trial in terms of the ability of the CRF1 receptor antagonist crinecerfont to reduce hydrocortisone dose while maintaining acceptable androstenedione levels?

Patricia Fechner, MD

What are the clinical translational implications of the results reported thus far from the CAHtalyst trial, and how might you identify patients in which crinecerfont would address unmet therapeutic need?

What are the clinical translational implications of the results reported thus far from the CAHtalyst trial, and how might you identify patients in which crinecerfont would address unmet therapeutic need?

Patricia Fechner, MD

Based on the mechanism-of-action of crinecerfont, and the results of the pediatric CAHtalyst trial, what role might this option provide for pediatric patients with CAH?

Based on the mechanism-of-action of crinecerfont, and the results of the pediatric CAHtalyst trial, what role might this option provide for pediatric patients with CAH?

Patricia Fechner, MD