by invitation only

Private sessions program

Friday 15 November

The speakers

Lunch

Lunch at Flendrigzaal

Welcome, introduction and aims

By H. Smeets / B. Verbrugge

Flendrigzaal

Clinical spectrum LAMA2 disease

By M. Durbeej-Hjalt / B. Verbrugge

Flendrigzaal

  • 13.15-13.45: A. Klein: Natural history MDC1A
  • 13.45-14.15: R. de Coo: Mild and atypical MDC1A
  • 14:15-14.45: H. Topaloglu: Clinical experience MDC1A
  • 14.45-15.10: D-J. and H. Stelwagen: The patient perspective
  • 15.10-15.35: M. Beare: The parent perspective (USA)
  • 15.35-16.05: A. Sarkozy: Congenital muscle disease
  • 16.05-16.15: Discussion, wrap-up and conclusions

Break

Trial readiness and therapeutic strategies (1)

By R. Foley / G. Dziewczapolski

Flendrigzaal

  • 16.30-17.00: G. Dziewczapolski: Clinical-Trial Readiness for LAMA2 disease
  • 17.00-17.30: N. Voermans/C. Erasmus: Towards Trial Readiness
  • 17.30-18.00: M. Durbeej: Compound-based therapy
  • 18.00-18.30: H. Sawnani: Preserving pulmonary function

Diner

By B. Verbrugge (Stichting Voor Sara)

Diner in Maastricht organised by Bram Verbrugge (Stichting Voor Sara)

Saturday 16 November

The speakers

Trial readiness and therapeutic strategies (2)

By M. Ruegg / A. Klein

Oak + Teak Room

  • 08.30-09.00: R. Foley: Clinical trials for MDC1A
  • 09.00-09.30: M. Girgenrath: Combinatorial therapy
  • 09.30-10.00: D. Kemaladewi: Genetic correction LAMA2 mutation in mouse model
  • 10.00-10.30: Discussion

MDC1A: The road to therapy

Open to all registrants. The complete programme can be found here.

Sunday 17 November

The speakers

Trial readiness and therapeutic strategies (3)

By H. Smeets / M. Beare

Pine Room

  • 08.30-09.00: P. Yurchenco: LAMA2 Linker Protein repair
  • 09.00-09.30: M. Rüegg: Protein replacement therapy MDC1A
  • 09.30-10.00: M. Sampaolesi: Mesoangioblast-therapy in animal models of muscular dystrophy
  • 10.00-10.30: S. Previtali: Mesoangioblast-therapy in a MDC1A mouse model

Break

Trial readiness and therapeutic strategies (4)

By H. Smeets / M. Beare
  • 11.00-11.30: F. Van Tienen: Muscle stem-cell in humans
  • 11.30-12.00: G. Cossu: Mesoangioblast-based therapy DMD
  • 12.00-13.00: H. smeets/B. Verbrugge: Discussion, wrap-up and conclusions

Sponsored Lunch - Meeting with Prothelia

Pine Room

  • 13.00-14.00: B. Hodges: Laminin-111 will transform LAMA2 deficiency

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