Rare Neurological Diseases: Spinal Muscular Atrophy and Huntington's Disease

Dr Peter Salgo, MD; Dr John Brandsema, MD; Sika Dunyoh; Dr Maria Lopes, MD, MS; and Dr Surya Singh, MD provide an overview of rare diseases, specifically spinal muscular atrophy (SMA) and Huntington

Tap into peer-to-peer dialogues covering different therapeutic areas in easy to digest videos, featuring managed care thought leaders.

Risdiplam and Final Thoughts
August 19, 2019 – 
The panel members review a pipeline medication, risdiplam, and provide some final thoughts on the future of spinal muscular atrophy (SMA) treatment.
Zolgensma and the START Trial for SMA
August 19, 2019 – 
Experts discuss the potential approval of Zolgensma (onasemnogene abeparvovec-xioi) and the START trial.
Payer Perspective and Value of Treatment of SMA
August 12, 2019 – 
Surya Singh, MD, provides a payer perspective on the value of treatment for patients with SMA.
Challenges of Payer Criteria and Cost of Therapy for SMA
August 12, 2019 – 
The panel shares challenges providers have based on payer criteria and the impact of cost of therapy.
Knowledge Among Payers and Payer Requirements for Rare Disease
August 05, 2019 – 
Surya Singh, MD, shares his thoughts on the knowledge payers have regarding rare diseases and the requirements for approval.
ENDEAR Study, Patient Factors, and Drug Approval Criteria
August 05, 2019 – 
Panelists share data from the ENDEAR study, including patient factors for its use, and also provide insights on the drug approval criteria.
Approval of Nusinersen for SMA
July 29, 2019 – 
John Brandsema, MD, introduces us to nusinersen, an FDA-approved treatment option for spinal muscular atrophy.
Treatment Goals and Multidisciplinary Management of SMA
July 29, 2019 – 
Experts review the goals of treatment for patients with spinal muscular atrophy and the multidisciplinary management of symptoms.
Economic Burden and Challenges of Trial of SMA
July 22, 2019 – 
The panel shed a light on the economic burden of spinal muscular atrophy and the challenges of trial design.
Diagnostic Tests and Scales of SMA
July 22, 2019 – 
John Brandsema, MD, discusses the diagnostic process for spinal muscular atrophy, including the different functional motor scales used to measure patient function.
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