1 u. 3 min.

Robin Lachmann, MD, PhD / Melissa Wasserstein, MD - Assessing the Potential Role of Emerging Therapies in the Early Diagnosis and Optimal Management of Chronic Visceral Acid Sphingomyelinase Deficiency PeerView Endocrinology & Diabetes CME/CNE/CPE Video Podcast

    • Wetenschap

Go online to PeerView.com/NPH860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. Chronic visceral acid sphingomyelinase deficiency (ASMD), also referred to as Niemann-Pick disease type B, is a rare and progressive autosomal recessive lysosomal storage disorder that causes progressive accumulation of sphingomyelin and other lipids in tissues throughout the body and is associated with significant morbidity and reduced life expectancy. The diagnosis of chronic visceral ASMD is often delayed by months or years, because its complex signs and symptoms overlap with other diseases. In this activity, leading experts review current consensus recommendations for the diagnosis of ASMD in pediatric and adult patients. The faculty also review the latest safety, efficacy, and tolerability data on emerging therapies and explore patient cases to offer practical strategies on how to optimally manage patients with chronic visceral ASMD. Upon completion of this activity, participants should be better able to: Diagnose individuals with ASMD through the application of recent consensus recommendations, Cite available efficacy, safety, and tolerability data on emerging therapeutic modalities for the treatment of patients with ASMD, Examine the potential impact that ongoing clinical trials of emerging therapeutic approaches may have on the future treatment of patients with ASMD.

Go online to PeerView.com/NPH860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. Chronic visceral acid sphingomyelinase deficiency (ASMD), also referred to as Niemann-Pick disease type B, is a rare and progressive autosomal recessive lysosomal storage disorder that causes progressive accumulation of sphingomyelin and other lipids in tissues throughout the body and is associated with significant morbidity and reduced life expectancy. The diagnosis of chronic visceral ASMD is often delayed by months or years, because its complex signs and symptoms overlap with other diseases. In this activity, leading experts review current consensus recommendations for the diagnosis of ASMD in pediatric and adult patients. The faculty also review the latest safety, efficacy, and tolerability data on emerging therapies and explore patient cases to offer practical strategies on how to optimally manage patients with chronic visceral ASMD. Upon completion of this activity, participants should be better able to: Diagnose individuals with ASMD through the application of recent consensus recommendations, Cite available efficacy, safety, and tolerability data on emerging therapeutic modalities for the treatment of patients with ASMD, Examine the potential impact that ongoing clinical trials of emerging therapeutic approaches may have on the future treatment of patients with ASMD.

1 u. 3 min.

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