The clinical trials and databases team includes three groups.
One group, led by Dr Odile Boespflug-Tanguy, is in charge of interventional research and manages clinical trials in children. Located at the Trousseau Pediatric Hospital in the I-Motion Institute, this team is composed of 3 more doctors, 8 CRAs, 4 physiotherapists, 2 managers, an occupational therapist, a quality engineer, an executive assistant and a secretaries (access to the composition of the Trousseau team and its recent publications).
The other group, specialised in non-interventional research, is in charge of databases and registries. Led by Dr Karim Wahbi, the group has another doctor and five clinical research associates (CRAs). This team is located in the Babinski building at the La Pitié-Salpêtrière Hospital.
Composition of the Salpêtrière team
Karim Wahbi, cardiologist
Rabah Ben Yaou, neurologist
Khadija Chikhaoui, CRA
Nawal Berber, CRA
Nadgib Taouagh, CRA
Néjette Lallouche, ARC
Recent publications
- Tosserams A, Papadopoulos C, Jardel C, Lemière I, Romero NB, De Lonlay P, Wahbi K, Voermans N, Hogrel JY, Laforêt P. Two new cases of mitochondrial myopathy with exercise intolerance, hyperlactatemia and cardiomyopathy, caused by
recessive SLC25A4 mutations. Mitochondrion. 2017 Aug 18. pii:S1567-7249(16)30193-3. doi: 10.1016/j.mito.2017.08.009. [Epub ahead of print]PubMed PMID: 28823815. - Heller F, Dabaj I, Mah JK, Bergounioux J, Essid A, Bönnemann CG, Rutkowski A, Bonne G, Quijano-Roy S, Wahbi K. Cardiac manifestations of congenital LMNA-related muscular dystrophy in children: three case reports and recommendations for care. Cardiol Young. 2017 Aug;27(6):1076-1082. doi:10.1017/S1047951116002079. Epub 2016 Dec 12. PubMed PMID: 27938454.
- Fayssoil A, Laforêt P, Bougouin W, Jardel C, Lombès A, Bécane HM, Berber N,Stojkovic T, Béhin A, Eymard B, Duboc D, Wahbi K. Prediction of long-term prognosis by heteroplasmy levels of the m.3243A>G mutation in patients with the mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes syndrome. Eur J Neurol. 2017 Feb;24(2):255-261. doi: 10.1111/ene.13176. Epub 2016 Nov 21. PubMed PMID: 27869334.
- Decostre V, Laforêt P, Nadaj-Pakleza A, De Antonio M, Leveugle S, Ollivier G, Canal A, Kachetel K, Petit F, Eymard B, Behin A, Wahbi K, Labrune P, Hogrel JY. Cross-sectional retrospective study of muscle function in patients with glycogen storage disease type III. Neuromuscul Disord. 2016 Sep;26(9):584-92. doi:10.1016/j.nmd.2016.06.460. Epub 2016 Jun 28. PubMed PMID: 27460348.
- Wahbi K, Bougouin W, Béhin A, Stojkovic T, Bécane HM, Jardel C, Berber N, Mochel F, Lombès A, Eymard B, Duboc D, Laforêt P. Long-term cardiac prognosis and risk stratification in 260 adults presenting with mitochondrial diseases. Eur Heart J. 2015 Nov 7;36(42):2886-93. doi: 10.1093/eurheartj/ehv307. Epub 2015 Jul 29. PubMed PMID: 26224072.
- Preisler N, Laforêt P, Madsen KL, Husu E, Vissing CR, Hedermann G, Galbo H, Lindberg C, Vissing J. Skeletal muscle metabolism during prolonged exercise in Pompe disease. Endocr Connect. 2017 Aug;6(6):384-394. doi: 10.1530/EC-17-0042. Epub 2017 May 10. PubMed PMID: 28490439.
- Papadopoulos C, Orlikowski D, Prigent H, Lacour A, Tard C, Furby A, Praline J, Solé G, Hogrel JY, De Antonio M, Semplicini C, Deibener-Kaminsky J, Kaminsky P, Eymard B, Taouagh N, Perniconi B, Hamroun D, Laforêt P; French Pompe Study Group. Effect of enzyme replacement therapy with alglucosidase alfa (Myozyme®) in 12 patients with advanced late-onset Pompe disease. Mol Genet Metab. 2017 Jun 20. pii: S1096-7192(17)30336-0. doi: 10.1016/j.ymgme.2017.06.007. [Epub ahead of print] PubMed PMID: 28648663.
- Byrne BJ, Geberhiwot T, Barshop BA, Barohn R, Hughes D, Bratkovic D, Desnuelle C, Laforet P, Mengel E, Roberts M, Haroldsen P, Reilley K, Jayaram K, Yang K, Walsh L; POM-001/002 Investigators. A study on the safety and efficacy of reveglucosidase alfa in patients with late-onset Pompe disease. Orphanet J Rare Dis. 2017 Aug 24;12(1):144. doi: 10.1186/s13023-017-0693-2. PubMed PMID: 28838325; PubMed Central PMCID: PMC5571484.
- van der Ploeg AT, Kruijshaar ME, Toscano A, Laforêt P, Angelini C, Lachmann RH, Pascual Pascual SI, Roberts M, Rösler K, Stulnig T, van Doorn PA, Van den Bergh PYK, Vissing J, Schoser B; European Pompe Consortium. European consensus for starting and stopping enzyme replacement therapy in adult patients with Pompe disease: a 10-year experience. Eur J Neurol. 2017 Jun;24(6):768-e31. doi:10.1111/ene.13285. Epub 2017 May 6. PubMed PMID: 28477382.
