Guest: Caroline Hastings, MD 


Guest: Raymond Y. Wang, MD 



  Niemann-Pick disease type C (NPC) is an ultra-rare, inherited, neurodegenerative disease with life-threatening complications. Due to its highly variable onset of neurological symptoms throughout one’s lifespan, recognizing and establishing diagnosis for NPC can be challenging for any clinician. That’s why Drs. Caroline Hastings and Raymond Y. Wang join Dr. Jennifer Caudle to help address those challenges with a detailed look at this genetic condition, the factors underlying its variable onset and course, and strategies to improve recognition, cross-specialty communications, and time to diagnosis.  Copyright ©2021 Orphazyme A/S. All rights reserved.NP-USA-1492-v1ORPHAZYME is a trademark or registered trademark of Orphazyme A/S.

Closing Gaps in the Recognition & Detection of Niemann-Pick Disease Type C

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  Guest: Richard S. Isaacson, MD 



  
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Host: Barry A. Singer, MD 



  Patients living with MS can experience hidden or invisible symptoms that may not be apparent to others but can affect how they feel and function. In this podcast, Dr. Barry Singer, the Director at the Multiple Sclerosis Center for Innovations in Care, and Rosario, a patient who has been living with MS for 10 years, discuss their clinical and personal experiences with MS, and the impact that MS can have on a patient's QoL. In the 2-year AFFIRM pivotal trial: Relapsing MS patients received 300 mg TYSABRI every 28 days (n=627) or placebo (n=315). Patients with PPMS, SPMS, and PRMS were excluded.2,3 83% of patients taking TYSABRI had no sustained disability progression for 12 weeks vs 71% with placebo (primary endpoint: 17% vs 29%; p<0.001).2,3 Dr. Singer reviews the results of a recent publication from Hersh et al, which describes the effect of TYSABRI® (natalizumab) on various aspects of a patient’s QoL. In the study, 164 patients reported their experience after starting TYSABRI using a standard Quality of Life in Neurological Disorders (Neuro-QoL) assessment of 12 domains, which included anxiety, depression, positive affect and well-being, emotional and behavioral dyscontrol, stigma, cognitive function, sleep disturbance, fatigue, participation and satisfaction with social …

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Host: Jacqueline Nicholas, MD, MPH 



  Ever had a multiple sclerosis (MS) patient who needed to start a DMT? Listen to Dr. Jacqueline Nicholas, System Chief of Neuroimmunology and Director of MS Research at Ohio Health, and Kim, a patient who has been living with MS for seven years, share their experiences with the treatment decision process, including benefit-risk considerations, treatment goals, and patient preferences. They talk about the process for starting and continuing treatment with TYSABRI® (natalizumab) and how Biogen Support Services can help. Please see Important Safety Information, including Boxed Warning for PML, below.  

Closing Gaps in the Recognition & Detection of Niemann-Pick Disease Type C

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