在不能行走的2型和3型脊髓性肌萎缩症患者中,相比于安慰剂,评价RO7034067在运动、呼吸等方面的有效性;了解是否会改善疾病对患者和家庭的影响;考察RO7034067的药代动力学特征、PK/PD关系以及其安全性特性。
序号 |
机构名称 |
主要研究者 |
国家 |
省(州) |
城市 |
1 |
UZ Leuven Gasthuisberg |
Nathalie Goemans |
比利时 |
Leuven |
Leuven |
2 |
Hopital Femme Mere Enfant; Medecine Physique et Readaptation Pediatrique – L’ESCALE |
Carole Vuillerot |
法国 |
Lyon |
Bron |
3 |
Hopital Armand Trousseau |
Laurent Servais |
法国 |
Paris |
Paris |
4 |
Universitatsklinikum Freiburg; Klinik fur Neuropadiatrie und Muskelerkrankungen |
Sabine Borell |
德国 |
Freiburg |
Freiburg |
5 |
Policlinico Agostino Gemelli; Dipartimento di Neuropsichiatria Infantile |
Eugenio Mercuri |
意大利 |
Lazio |
Roma |
6 |
Fondazione IRCCS Istituto Neurologico “Carlo Besta”; UO di Neurologia dello Sviluppo |
Riccardo Masson |
意大利 |
Lombardia |
MILANO |
7 |
National Center Of Neurology And Psychiatry Hospital |
Hirofumi Komaki |
日本 |
Tokyo |
Tokyo |
8 |
Jichi Medical University Hospital |
Hitoshi Osaka |
日本 |
Tochigi |
Tochigi |
9 |
Center Hospital of the National Center for Global Health and Medicine |
Kayoko Saito |
日本 |
Tokyo |
Tokyo |
10 |
Miyagi Children's Hospital |
Kazuhiro Haginoya |
日本 |
Miyagi |
Miyagi |
11 |
Shiga Medical Center for Children |
Minoru Shibata |
日本 |
Shiga |
Shiga |
12 |
Hiroshima University Hospital |
Nobutsune Ishikawa |
日本 |
Hiroshima |
Hiroshima |
13 |
Minami Kyushu National Hospital |
Nozomi Sano |
日本 |
Kagoshima |
Kagoshima |
14 |
Fukuoka Children's Hospital |
Ryutaro Kira |
日本 |
Fukuoka |
Fukuoka |
15 |
Shizuoka Children's Hospital |
Seiji Watanabe |
日本 |
Shizuoka |
Shizuoka |
16 |
Hyogo College of Medicine Hospital |
Yasuhiro Takeshima |
日本 |
Hyogo |
Hyogo |
17 |
北京大学第一医院 |
熊晖 |
中国 |
北京 |
北京 |
18 |
复旦大学附属儿科医院 |
王艺 |
中国 |
上海 |
上海 |
19 |
CHR DE LA CITADELLE |
Aurore Daron |
比利时 |
Liege |
Liege |
20 |
UZ Gent |
Nicolas Deconinck |
比利时 |
Gent |
Gent |
21 |
INSTITUTO DE PUERICULTURA E PEDIATRIA MARTAGAO GESTEIRA |
Alexandra Prufer |
巴西 |
RJ |
Rio de Janeiro |
22 |
London Health Sciences Centre; Children's Hospital; Pediatrics |
Craig Campbell |
加拿大 |
Ontario |
London |
23 |
Alberta Children's Hospital Division of Pediatric Neurology |
Jean Mah |
加拿大 |
Alberta |
Calgary |
24 |
McGill University Health Center |
Maryam Oskoui |
加拿大 |
Quebec |
Montreal |
25 |
Clinical Medical Center Zagreb; University Hospital Rebro Department of Paediatrics |
Nina Barisic |
克罗地亚 |
ZAGREB |
ZAGREB |
26 |
CHRU de Montpellier, Hopital Gui de Chauliac; Service de Neuropediatrie |
Francois Rivier |
法国 |
Montpellier |
Montpellier |
27 |
Hopital Necker-Enfants Malades; Service de neuropediatrie |
Isabelle Desguerre |
法国 |
PARIS |
PARIS |
28 |
Hopital Roger Salengro |
Jean Marie Cuisset |
法国 |
Lille |
Lille |
29 |
CHU de Nantes - Hotel Dieu |
Yann Pereon |
法国 |
NANTES |
NANTES |
30 |
IRCCS Istituto Giannina Gaslini; U.O.S.D. Centro di Miologia e Patologie Neurodegenerative |
Claudio Bruno |
意大利 |
Liguria |
Genova |
31 |
IRCCS Ospedale Pediatrico Bambino Gesu; U.O. Malattie Neuromuscolari e Neurodegenerative |
Enrico Bertini |
意大利 |
Lazio |
Roma |
32 |
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Unità Operativa Complessa di Neurologia |
Giacomo Pietro Comi |
意大利 |
Lombardia |
Milano |
33 |
Azienda Ospedaliera Universitaria Policlinico G.Martino; Dip. Neurologia e Malattie neuromuscolari |
Giuseppe Vita |
意大利 |
Sicilia |
Messina |
34 |
Klinika Neurologii I Wydzialu Lekarskiego WUM w Warszawie |
Anna Kostera-Pruszczyk |
波兰 |
WARSZAWA |
WARSZAWA |
35 |
Szpital Kliniczny im. H. Swiecickiego U.M. w Poznaniu; Od. Kliniczny Neurologii Dzieci i M?odziezy |
BarbaraSteinborn |
波兰 |
Poznan |
Poznan |
36 |
Szpital Gdanskiego Uniwersytetu Medycznego; Clinic of developmental neurology |
Maria Mazurkiewicz-Be?dzinska |
波兰 |
Gdansk |
Gdansk |
37 |
Russian Children Neuromuscular Center of Veltischev |
Dmitry Vlodavets |
俄罗斯 |
MOSKOVSKAJA OBLAST |
Moscow |
38 |
Clinic for Neurology and Psychiatry for Children and Youth |
Vedrana Milic-Rasic |
塞尔维亚 |
Belgrade |
Belgrade |
39 |
Hospital Sant Joan De Deu |
Andres Nascimento Osorio |
西班牙 |
BARCELONA |
BARCELONA |
40 |
Hospital Universitari Vall d'Hebron; Servicio de Reumatologia |
Francina Munell Casadesus |
西班牙 |
Barcelona |
Barcelona |
41 |
Hospital Universitario Virgen del Rocio; Servicio de Neuropediatra |
Marcos Madruga |
西班牙 |
Sevilla |
Sevilla |
42 |
Hospital Universitario La Paz |
Samuel Pascual Pascual |
西班牙 |
Madrid |
Madrid |
43 |
Hacettepe University, School of Medicine; Pediatrics Department; Pediatrics Child Neurology Unit |
Haluk Topaloglu |
土耳其 |
Ankara |
Ankara |
44 |
Columbia University Medical Center; The Neurological Institute of New York |
Claudia Chiriboga |
美国 |
New York |
New York |
45 |
Stanford University Medical Center |
John Day |
美国 |
California |
Palo Alto |