PROGRAM

Sunday June 9, 2019

1. MEASURING THERAPEUTIC SUCCESS:
  Chairs: M.Schmuth, J.Bauer
12:00 - 12:30 Systemic complications in genodermatoses. - C.Bodemer
12:30 - 13:00 Melanoma and squamous cell carcinoma in Ichthyosis and EB. - R.Gruber and M.Laimer
13:00 - 13:30 Quality of life in genodermatoses. - M.El Hachem
 
Outcome Measures
 
13:30 - 15:00 Consensus Workshop 1 - Subgroup EB. - D.Murell (Coordinator)
  13:30 - 13:45      Overview of EB outcome validation. - D.Murell
  13:45 - 14:00      EB outcome measures in use in clinical trials. - H.Rishel
  14:00 - 14:15      IGA scores for EBS. - P.Zografos
  14:15 - 14:25      Blister counts in diacerein trial for EBS. - V.Wally
  14:25 - 14:35      Wound monitoring using photographic systems in RDEB gene therapy trials. - P.Marinkovich
  14:35 - 14:45      EB oropharyngeal severity score (EBOS). - J.Salas
  14:45 - 15:00      Discussion
13:30 - 15:00 Consensus Workshop 2 - Subgroup Ichthyoses. - K.Choate (Coordinator)
  13:30 - 13:45      Introduction. - K.Choate
  13:45 - 14:00      Liarozol experience. - H.Traupe
  14:00 - 14:15      Ichthyosis severity assessment tool. - S.H.Rabia
  14:15 - 14:30      Visual index for Ichthyosis severity. - K.Choate
  14:30 - 15:00      Discussion. - K.Choate
15:00 - 16:00 Results/Plenary discussion. - L.Bruckner-Tuderman, H.Traupe (Moderators)
 
16:00 - 16:30 Break and Poster
 
  Chairs: K.Tamai, F.Palisson
16:30 - 17:00 Developing an ATMP for a rare disease - Regulatory experience. - I.Reischl
17:00 - 17:30 Perturbation biology and bioinformatics for N-of-1 trials. - Z.Trajanoski
17:30 - 17:45 Trial design for rare diseases - the patient´s perspective. - S.Kitzmüller
17:45 - 18:00 1. Complex dental treatment in children with dystrophic epidermolysis bullosa. -
  2. Oral manifestation of KID syndrome. - M.Korolenkova (1 + 2)
 
18:00 - 18:30 Break and Posters
 
2. THERAPEUTIC ADVANCES - PRECLINICAL:
  Chairs: P.Steijlen, E.Sprecher
18:30 - 19:00 Peptides. - A.Nyström
19:00 - 19:30 Role of inflammation in Ichthyoses. - A.Paller
19:30 - 20:15 Key lecture: Identifying the molecular mechanisms of epidermal stem cell aging. - G.Solanas
 
20:15 - 23:00 Dinner, Wine and Poster

Monday June 10, 2019

3. THERAPEUTIC ADVANCES - CLINICAL TRIALS:
  Chairs: J.Fischer, Y.Yang
08:00 - 08:20 Small molecules. - V.Wally
08:20 - 08:40 Novel systemic therapies for Ichthyoses: clinical experiences in Münster. - V.Oji
08:40 - 09:10 Nucleases. - F.Larcher
09:10 - 09:50 Gene replacement. - M.De Luca
 
09:50 - 10:10 Break
 
  Chairs: M.Laimer, R.Gruber
10:10 - 10:40 EBgraft for RDEB. - A.Hovnanian
10:40 - 11:25 In vivo molecular corrective therapies - P.Marinkovich
11:25 - 11:55 TGM1 Supplementation in ARCI: Patient involvement in research. - UNITI (and Calheiros)
 
12:00 Farewell and Departure
Logo - Krystal
Logo - Castle Creek Pharma