Dr Maria Vanegas shares the significance of Outcomes Beyond Motor Measures:

• Understand what outcomes we currently measure and explore what (else) we should consider measuring.
• Explore outcomes prioritised by patients and caregivers: Patient-reported outcome measures (PROMs).
• Identify and address gaps in clinical practice, to ensure patients go from survival to thriving.

Professor Giovanni Baranello shares the latest retrospective assessment of feeding and nutrition after 2 years of Evrysdi treatment in younger children with SMA using the CEDAS.

• Understand what the Children’s Eating and Drinking Activity Scale (CEDAS) is and how it can be used as a proxy to assess swallowing and feeding function.
• Understand how CEDAS data is expected to provide further context to feeding and swallowing function data in younger children treated with Evrysdi.
• Explore the outcomes of the independent retrospective CEDAS scoring of FIREFISH and RAINBOWFISH studies.

Dr Channa Hewamadduma explores what the latest global real world evidence (RWE) shows about Evrysdi in the treatment of SMA:

• Explore the main findings on the efficacy and safety profile of Evrysdi from the latest global real world evidence (RWE).
• Understand how RWE can compliment clinical trial data in the treatment of SMA.
• Understand how RWE can be used to tailor treatment decisions.
• Explore how RWE data collection for SMA may evolve in the UK.

Jane O’Connor, a Neuromuscular Care Advisor, and Alisha Hussain, a patient living with SMA Type 3, discuss:

• Alisha’s experience of navigating key life transitions and how these transitions affected her wellbeing, independence and quality of life.
• The changes, resources and innovations in healthcare that Alisha feels could better support people with SMA during these life transitions.

Neuromuscular Care Advisor, Jane O’Connor and Neuromuscular Transition Nurse Specialist, Lisa Cutsey, discuss:

• Different life transitions a person living with SMA might face and how HCPs can provide optimal support.
• How patients needs and goals should be incorporated into transition journey planning.
• Advice to improve how transitions are managed for your current and future patients.

Professor Laurent Servais shares the latest Evrysdi data from SUNFISH Parts 1 and 2: 5-Year Efficacy and Safety Data of Evrysdi in Types 2 and 3 SMA:

• Understand the importance of early access to treatment for SMA.
• Explore the motor function outcomes at the end of the the 5-year SUNFISH studies.
• Understand the patient and caregiver perspective, to determine overall impact of treatment on daily life.

Professor Neil Hawkins shares an indirect treatment comparison (ITC),‘The Long-Term Comparative Efficacy and Safety Profile of Evrysdi versus Nusinersen in Children with Type 1 SMA’:

• Understand what a matching- adjusted indirect comparison (MAIC) is.
• Understand the key results from the MAIC of Evrysdi (FIREFISH) compared with Nusinersen (ENDEAR/SHINE).
• Understand why in the absence of head-to-head trials, the ITC was conducted and explore possible limitations and how to interpret these results.

Disclaimer: MAIC studies, while informative, are based on statistical modeling and indirect comparisons. They should be interpreted with caution as they are not a substitute for direct head-to-head clinical trials. Individual patient outcomes may vary.

Advanced Paediatric Nurse Practitioner in Neuromuscular Therapies, Nicholas Mills and Paediatric Neuromuscular Physiotherapist, Sarah D’Urso explore real case studies of patients who have switched treatments:

• Understand the reasons patients may have had for changing treatments.
• Understand the importance of early and continuous patient dialogues to help manage expectations and ensure treatment is optimised.
• Explore how the wider multidisciplinary team can best manage conversations with patients, to ensure individuals are on the treatment that best suits their needs and lifestyle.

Paediatric Neuromuscular Clinical Nurse Specialist, Natalie Smith, discusses challenges around misinformation amongst the SMA community and shares how Healthcare Professionals can be empowered to manage conversations around misinformation:

• Understand what misinformation is, why it arises in the SMA community and examples of misinformation related to Evrysdi that has been raised by patients.
• Understand key strategies to support patients with SMA and their families after a new diagnosis.
• Explore ways we can empower the multidisciplinary team to confidently manage conversations with patient and caregivers about misinformation.

Dr Channa Hewamadduma explores the latest evidence around Fertility in patients living with SMA:

• Understand the latest evidence on the impact of SMA on fertility.
• Understand the latest evidence on the effect of Evyrsdi on fertility.
• Explore the key considerations on fertility for individuals living with SMA.