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Times are shown in UK time zone. Programme and speakers may be subject to change.


November 7


Day one

17:00            Introduction – Jarod Wong & Elizabeth Vroom

The impact of steroids and DMD on musculoskeletal and endocrine outcomes

(Chairs: Pat Furlong & Jarod Wong)


17:05           
Bone & endocrine morbidity in DMD with focus on the 2018 minimum international standards of care. David Weber (Philadelphia, USA)

17:30             Patient story: Fat embolism syndrome

17:35       Steroid regimen and impact on muscle-bone & endocrine morbidity: Results from the FOR-DMD clinical trial. Michela Guglieri (Newcastle, UK) & Leanne Ward (Ottawa, Canada)

18:00       High dose weekend only steroid regimen in DMD.  Anne Connolly (Columbus, USA)

18:25       Patient story: Painful vertebral fracture

18:30       Discussion
All, moderated by Jarod Wong

18:45       Break

Implementing standards of care in the clinic with focus on bone/endocrine monitoring and management: Challenges, successes and lessons learnt

(Chairs: Alex Johnson & Michela Guglieri)

18:50       World Duchenne Organization and its role in implementation of standards of care globally. Elizabeth Vroom (Representing World Duchenne Organization)

18:55       The Parent Project Muscular Dystrophy certified centres approach in the USA. Rachel Schrader (Representing Parent Project Muscular Dystrophy)

19:00       The Duchenne Centre Netherlands approach in the Netherlands. Erik Niks (Leiden, Netherlands)

19:05       The DMD Care UK approach in the UK NorthStar network and a national health system. Cathy Turner (Newcastle, UK representing DMD Care UK/Duchenne UK)

19:10       Discussion
All, moderated by Michela Guglieri

Current bone monitoring and treatment strategies in DMD and the 2018 international standards of care

(Chairs: Elizabeth Vroom & Leanne Ward)

19:15       A walk-through of paediatric radiographic vertebral fracture evaluation
Khaldoun Koujak (Ontario, Canada)

19:40     Patient story: Reaction to bisphosphonate (5 min)

19:45     An appraisal of the published literature on bone protective therapies in DMD recommended in the 2018 standard of care. Craig Munn (Brisbane, Australia)

20:10       Discussion
All moderated by Leanne Ward

20:30       Summary and close 
Jarod Wong


November 14


Day two

17:00       Introduction
Jarod Wong & Elizabeth Vroom

17:05       Summary of DMD related abstracts from the International Conference on Children’s Bone Health, Dublin 2022. Frank Rauch (Montreal, Canada)

Moving beyond the 2018 minimum international standards of care for bone management in DMD

Co-chairs: Leanne Ward & Michela Guglieri

17:15       Significant bone loss following loss of ambulation in DMD.  Nicola Crabtree (Birmingham, UK)

17:35       The vertebral fracture cascade in DMD: Should the treatment threshold with bisphosphonates be reconsidered? Jarod Wong (Glasgow, UK)

17:55       Clinical predictors for vertebral fracture in DMD Kim Phung (Ottawa, Canada)

18:15       Oral bisphosphonate therapy in DMD with mild vertebral fracture or declining bone density. Brenda Wong (Worcester, USA)

18:35       The patient’s perspective on the 2018 standards of care of osteoporosis management in DMD. Elizabeth Vroom (Duchenne Parent Project Netherlands), Pat Furlong (Parent Project Muscular Dystrophy), Alex Johnson (Duchenne UK)

18:45       Discussion
All, moderated by Leanne Ward & Michela Guglieri

19:00       Break (5 min)

New therapeutic approaches for osteoporosis in DMD

Co chairs: Jarod Wong & Michela Guglieri

19:05      RANKL inhibition treatment and impact on musculoskeletal outcomes in an animal model of DMD. Soher Jayash (Edinburgh, UK)

19:25       The role of Denosumab for management of osteoporosis in DMD. Leanne Ward (Ottawa, Canada)

19:45       Vamorolone: Impact on skeletal muscle and bone outcome. Eric Hoffman (Binghamton, USA) and Stefan Jackowski (Ottawa, Canada)

20:05       Discussion
All, moderated by Michela Guglieri & Jarod Wong

20:20       Summary and close
Jarod Wong/Elizabeth Vroom