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- Spinal Muscular Atrophy: Update in Best Clinical Care and Genetic Therapies
Spinal Muscular Atrophy: Update in Best Clinical Care and Genetic Therapies
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🗓️ Event Details
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📅 Date: September 10, 2025
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⏰ Time: 6:00 PM CET
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⏳ Duration: 90 Minutes
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🌐 Language: English
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💰 Cost: Free
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📝 Self-Assessment Activity: Not available
Title: Spinal Muscular Atrophy: Update in Best Clinical Care and Genetic Therapies
Speakers: Tawfeg Ben-Omran; Chahnez Charfi Triki; Ramesh Konanki; Juliana Gurgel; Ann Mathew
When: September 10, 2025
Time: 6:00 PM CET
About the Talk
Speaker: Tawfeg Ben-Omran
Title: Spinal Muscular Atrophy: Update in Best Clinical Care and Genetic Therapies
About Topic:
This talk provides a concise overview of the clinical and genetic aspects of SMA, along with updates on current genetic therapies and real-world treatment experiences. It emphasizes the critical importance of early diagnosis and intervention—best achieved through implementation of newborn screening programs.
Learning Objectives:
• Understand the genetic and clinical presentation of SMA
• Review management strategies using a multidisciplinary approach
• Explore novel therapies and therapeutic strategies in real-world practice
Speaker: Ann Mathew
Title: “Beyond drugs: multidisciplinary care that changes trajectories”
- Respiratory protocols, nutrition, physio/OT, orthopaedics, scoliosis pathways.
- Home-based care models, tele-rehab, caregiver training, assistive tech.
- Outcome tracking that supports reimbursement and quality improvement.
Speaker: Ramesh Konanki
Title: “Closing the diagnostic gap: triage algorithms, confirmatory testing, and referral networks”
- Sensible early-signs triage for primary care.
- Affordable confirmatory testing options; lab turn-around targets.
- Building state/regional hubs; leveraging government schemes & CSR.
Speaker: Juliana Gurgel
Proposed Title: “From pilot to policy: integrating SMA into newborn screening and national formularies”
- Brazil pathway: pilot data → scale-up; payer negotiations; centre designation.
- Managing therapy logistics (intrathecal vs oral vs vector-based) in a large federation.
- Data for advocacy: outcomes, safety, and budget impact
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