by invitation only

Private sessions program

Friday 15 November

The speakers

12.00 - 13.00

Lunch

Lunch at Flendrigzaal

13.00 - 13.15

Welcome, introduction and aims

By H. Smeets / B. Verbrugge

Flendrigzaal

13.15 - 16.15

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
16.15 - 16.30

Break

16.30 - 18.30

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
19.00

Diner

By B. Verbrugge (Stichting Voor Sara)

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

Saturday 16 November

The speakers

08.30 - 10.30

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
10.30 - 19.30

MDC1A: The road to therapy

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

Sunday 17 November

The speakers

08.30 - 10.30

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
10.30 - 11.00

Break

11.00 - 13.00

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
13.00 -14.00

Sponsored Lunch - Meeting with Prothelia

Pine Room

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

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