Managing Spinal Muscular Atrophy in Adult Patients

Key opinion leaders offer a concise summary of the clinical characteristics and classifications of spinal muscular atrophy (SMA), emphasizing the distinctive features observed in adult patients with SMA.

Panelists examine the natural history of spinal muscular atrophy (SMA), assessing the characteristic indicators of motor function deterioration and the loss of ambulatory abilities as patients advance in age.

Leading medical professionals specializing in spinal muscular atrophy (SMA) discuss the common clinical presentations of undiagnosed or newly diagnosed adult patients, highlighting crucial signs and symptoms that healthcare providers should recognize.

Key opinion leaders (KOLs) address the challenges and obstacles associated with transitioning patients from pediatric to adult care as they age out of the pediatric system, offering valuable insights and strategies for successfully navigating this process.

Key opinion leaders (KOLs) further explore the transition of care from pediatric to adult healthcare systems for patients with spinal muscular atrophy (SMA), emphasizing the essential role of multidisciplinary support in ensuring a successful transition.

Clinical experts in spinal muscular atrophy (SMA) address the issue of patient disengagement from healthcare services, which can occur due to a perceived lack of disease progression or stabilization, leading to undetected disease advancement and increased risk to patients, and provide recommendations for encouraging patients to re-engage with medical care.

Key opinion leaders (KOLs) examine the mechanisms of action and other pertinent information regarding FDA-approved treatments for spinal muscular atrophy (SMA), sharing their perspectives on these therapies with a particular focus on the various modes of administration.

Panelists explore critical considerations when choosing a treatment approach for treatment-naïve adult patients with spinal muscular atrophy (SMA), taking into account factors such as disease severity, patient preferences, and potential treatment outcomes.

Key opinion leaders share their strategies for discussing potential changes in therapy and transitioning between treatments with patients, emphasizing the importance of reassuring patients that such changes are intended to provide long-term benefits for managing their spinal muscular atrophy.

Panelists share their approaches to counseling adult patients with spinal muscular atrophy (SMA) about treatment expectations.

Panelists delve into strategies for engaging in conversations with patients about defining and identifying successful therapy outcomes, providing guidance on how to effectively communicate and establish realistic expectations for treatment success in the context of spinal muscular atrophy (SMA).

Key opinion leaders address the scarcity of randomized controlled trial data for SMA treatments in adult populations, highlighting the availability of real-world data from numerous studies and provide guidance on how to incorporate this information when making treatment decisions for adult patients with spinal muscular atrophy.

Panelists examine the available real-world data (RWD) on the use of risdiplam in adult patients with spinal muscular atrophy (SMA) from various studies conducted worldwide, discussing its potential efficacy and safety as a treatment option for adult SMA patients.

Medical professionals explore the challenges and considerations associated with patients with spinal muscular atrophy (SMA) transitioning to independent living, emphasizing the importance of maintaining continuity of care and addressing potential adherence issues to ensure optimal disease management.