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Phyllis W. Speiser, MD

Can you briefly summarize how you developed your interest in CAH?

Can you briefly summarize how you developed your interest in CAH?

Phyllis W. Speiser, MD

What does the landscape look like for diagnosing, assessing, and monitoring persons—adult and pediatric—who are suspected of having or have confirmed CAH?

What does the landscape look like for diagnosing, assessing, and monitoring persons—adult and pediatric—who are suspected of having or have confirmed CAH?

Phyllis W. Speiser, MD

Can you provide a summary of the pathobiology and hormonal ramifications of the classic form of CAH caused by 21-OH deficiency, and the unmet need for therapies that address hormonal derangments?

Can you provide a summary of the pathobiology and hormonal ramifications of the classic form of CAH caused by 21-OH deficiency, and the unmet need for therapies that address hormonal derangments?

Phyllis W. Speiser, MD

What are the challenges of optimizing therapy—even with glucocorticoids—considering that in CAH the aldosterone, androgen, and glucocorticoid pathways are coupled with one another?

What are the challenges of optimizing therapy—even with glucocorticoids—considering that in CAH the aldosterone, androgen, and glucocorticoid pathways are coupled with one another?

Phyllis W. Speiser, MD

What hormones are being/should be monitored in persons with CAH to ensure that adequate suppression of the HPA axis has been achieved that is clinically meaningful?

What hormones are being/should be monitored in persons with CAH to ensure that adequate suppression of the HPA axis has been achieved that is clinically meaningful?

Phyllis W. Speiser, MD

Can you discuss the complications of excessive glucocorticoid exposure in children, adolescents, and adults, and the impact on cardiac and metabolic morbidities? What is the rational for looking for alternatives to glucocorticoid therapy for CAH?

Can you discuss the complications of excessive glucocorticoid exposure in children, adolescents, and adults, and the impact on cardiac and metabolic morbidities? What is the rational for looking for alternatives to glucocorticoid therapy for CAH?

Phyllis W. Speiser, MD

Can you discuss the CAHtalyst pediatric strategies aimed at understanding whether a de-coupling of the androstenedione and glucocorticoid pathways might be a solution for reducing the burden of glucocorticoid therapy?

Can you discuss the CAHtalyst pediatric strategies aimed at understanding whether a de-coupling of the androstenedione and glucocorticoid pathways might be a solution for reducing the burden of glucocorticoid therapy?

Phyllis W. Speiser, MD

Can you discuss what monitoring and therapeutic end points—i.e., glucocorticoid sparing—were selected, and why, for the CAHtalyst trial? And what were the trial’s primary objectives?

Can you discuss what monitoring and therapeutic end points—i.e., glucocorticoid sparing—were selected, and why, for the CAHtalyst trial? And what were the trial’s primary objectives?

Phyllis W. Speiser, MD

In phase 1 of the CAHtalyst pediatric study, did investigators demonstrate a statistically significant decrease in serum androstenedione from baseline with crinecerfont at Week 4 over baseline?

In phase 1 of the CAHtalyst pediatric study, did investigators demonstrate a statistically significant decrease in serum androstenedione from baseline with crinecerfont at Week 4 over baseline?

Phyllis W. Speiser, MD

What can you tell us about some of the key features of the CAHtalyst pediatric trial, including retention of trial subjects, and achieving androstenedione end points in patients on crinecerfont?

What can you tell us about some of the key features of the CAHtalyst pediatric trial, including retention of trial subjects, and achieving androstenedione end points in patients on crinecerfont?

Phyllis W. Speiser, MD

In the second phase of the CAHtalyst pediatric trial, can you discuss initiating down-titration of glucocorticoid therapy, and what levels of glucocorticoid sparing were targeted?

In the second phase of the CAHtalyst pediatric trial, can you discuss initiating down-titration of glucocorticoid therapy, and what levels of glucocorticoid sparing were targeted?

Phyllis W. Speiser, MD

Based on the CAHtalyst pediatric trial, were you able to down-titrate glucocorticoid doses in phase 2, evaluating the potential of crinecerfont to achieve glucocorticoid sparing in persons with CAH?

Based on the CAHtalyst pediatric trial, were you able to down-titrate glucocorticoid doses in phase 2, evaluating the potential of crinecerfont to achieve glucocorticoid sparing in persons with CAH?

Phyllis W. Speiser, MD

Based on the CAHtalyst pediatric trial, what subgroups of children and adults with classical CAH represent ideal patients for treatment with CRF1-targeting inhibitors such as crinecerfont?

Based on the CAHtalyst pediatric trial, what subgroups of children and adults with classical CAH represent ideal patients for treatment with CRF1-targeting inhibitors such as crinecerfont?

Phyllis W. Speiser, MD