Professor of clinical pediatrics and neurology; Haberfeld Family Endowed Chair in Pediatric Neuromuscular Disorders, Children’s Hospital Colorado
Role of Combination Therapies in SMA Management
John Brandsema, MD, and Julie Parsons, MD, review the evolution of combination therapy in clinical practice for the management of SMA.
SMA: Patient Adherence to Therapy
John Brandsema, MD, comments on the importance of patient compliance to therapy to manage spinal muscular atrophy.
Using Small Molecule Treatment for SMA Management
Experts in pediatric neurology share insights on the role of small molecule treatment for SMA through the recent FDA approval of risdiplam and discuss factors to consider when tailoring therapy for individual patients.
Role of Gene Transfer Therapy in SMA
John Brandsema, MD, leads the discussion on the concept of gene transfer for the management of SMA and the recent approval of onasemnogene abeparvovec-xioi.
Role of Nusinersen in SMA: Efficacy and Treatment Response
Experts in pediatric neurology provide insight on the efficacy of nusinersen and treatment response in patients with SMA.
Targeted Therapies for SMA Management: Nusinersen
John Brandsema, MD, and Julie Parsons, MD, discuss the approval of nusinersen for the treatment of spinal muscular atrophy and their personal experiences regarding drug safety and administration in patients.
Early Referral to Neurologists for SMA Diagnosis
Expert pediatric neurologists review the importance of early recognition and referral to neurologists for the diagnosis of spinal muscular atrophy and when it’s appropriate to approach treatment.
Educating Primary Care Physicians on the Diagnosis of SMA
Julie Parsons, MD, comments on the role of primary care providers and gene testing for the early diagnosis of spinal muscular atrophy.
Early Detection and Screening of Spinal Muscular Atrophy
Identifying spinal muscular atrophy through early diagnosis using prenatal and newborn screenings is discussed.
Managing SMA in Clinical Practice
Julie Parsons, MD, discusses the importance of a multidisciplinary team approach to managing SMA in clinical practice.
Classification of Spinal Muscular Atrophy (SMA)
John Brandsema, MD, leads the discussion on the 5 main types of spinal muscular atrophy and the evolution of disease classification.
Looking to the Future of SMA Treatment: Julie Parsons, MD
The Endowed Chair in Pediatric Neuromuscular Disorders and professor of clinical pediatrics and neurology, at Children’s Hospital Colorado discussed the future of SMA treatment.
A Rational Approach to Assessing SMA Therapies: Julie Parsons, MD
The primary investigator of the phase 4 RESPOND study discussed the role that postmarketing studies can play in assessing treatments for SMA.
Phase 4 RESPOND Trial and the Need for Similar Trials in SMA: Julie Parsons, MD
The primary investigator of RESPOND discussed the trial’s goals and measures, as well as insight into why clinical trials like it are important in spinal muscular atrophy.
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