Literature DB >> 17300938

Issues in SMA clinical trial design. The International Coordinating Committee (ICC) for SMA Subcommittee on SMA Clinical Trial Design.

P Kaufmann1, F Muntoni.   

Abstract

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Year:  2007        PMID： 17300938      PMCID： PMC3250230          DOI： 10.1016/j.nmd.2006.12.001

Source DB:  PubMed          Journal:  Neuromuscul Disord        ISSN： 0960-8966            Impact factor:   4.296


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  28 in total

1.  On the use of nonparametric curves in phase I trials with low toxicity tolerance.

Authors:  Ying Kuen Cheung
Journal:  Biometrics       Date:  2002-03       Impact factor: 2.571

2.  The Hammersmith functional motor scale for children with spinal muscular atrophy: a scale to test ability and monitor progress in children with limited ambulation.

Authors:  Marion Main; Harvey Kairon; Eugenio Mercuri; Francesco Muntoni
Journal:  Eur J Paediatr Neurol       Date:  2003       Impact factor: 3.140

3.  A simple technique to evaluate model sensitivity in the continual reassessment method.

Authors:  Ying Kuen Cheung; Rick Chappell
Journal:  Biometrics       Date:  2002-09       Impact factor: 2.571

4.  Optimal two-stage designs for phase II clinical trials.

Authors:  R Simon
Journal:  Control Clin Trials       Date:  1989-03

Review 5.  Neuroprotective agents for clinical trials in ALS: a systematic assessment.

Authors:  B J Traynor; L Bruijn; R Conwit; F Beal; G O'Neill; S C Fagan; M E Cudkowicz
Journal:  Neurology       Date:  2006-07-11       Impact factor: 9.910

6.  A randomized sequential trial of creatine in amyotrophic lateral sclerosis.

Authors:  G J Groeneveld; Jan H Veldink; Ingeborg van der Tweel; Sandra Kalmijn; Cornelis Beijer; Marianne de Visser; John H J Wokke; Hessel Franssen; Leonard H van den Berg
Journal:  Ann Neurol       Date:  2003-04       Impact factor: 10.422

7.  Sequential designs for phase I clinical trials with late-onset toxicities.

Authors:  Y K Cheung; R Chappell
Journal:  Biometrics       Date:  2000-12       Impact factor: 2.571

Review 8.  Concerns about the design of clinical trials for spinal muscular atrophy.

Authors:  Thomas O Crawford
Journal:  Neuromuscul Disord       Date:  2004-09       Impact factor: 4.296

9.  Outcome measures for pediatric spinal muscular atrophy.

Authors:  Susan T Iannaccone
Journal:  Arch Neurol       Date:  2002-09

10.  A randomized, placebo-controlled trial of topiramate in amyotrophic lateral sclerosis.

Authors:  M E Cudkowicz; J M Shefner; D A Schoenfeld; R H Brown; H Johnson; M Qureshi; M Jacobs; J D Rothstein; S H Appel; R M Pascuzzi; T D Heiman-Patterson; P D Donofrio; W S David; J A Russell; R Tandan; E P Pioro; K J Felice; J Rosenfeld; R N Mandler; G M Sachs; W G Bradley; E M Raynor; G D Baquis; J M Belsh; S Novella; J Goldstein; J Hulihan
Journal:  Neurology       Date:  2003-08-26       Impact factor: 9.910
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  7 in total

1.  Spinal muscular atrophy: advances in research and consensus on care of patients.

Authors:  Ching H Wang; Mitchell R Lunn
Journal:  Curr Treat Options Neurol       Date:  2008-11       Impact factor: 3.598

2.  Rasch analysis of clinical outcome measures in spinal muscular atrophy.

Authors:  Stefan J Cano; Anna Mayhew; Allan M Glanzman; Kristin J Krosschell; Kathryn J Swoboda; Marion Main; Birgit F Steffensen; Carole Bérard; Françoise Girardot; Christine A M Payan; Eugenio Mercuri; Elena Mazzone; Bakri Elsheikh; Julaine Florence; Linda S Hynan; Susan T Iannaccone; Leslie L Nelson; Shree Pandya; Michael Rose; Charles Scott; Reza Sadjadi; Mackensie A Yore; Cynthia Joyce; John T Kissel
Journal:  Muscle Nerve       Date:  2013-07-26       Impact factor: 3.217

3.  Clinical and molecular cross-sectional study of a cohort of adult type III spinal muscular atrophy patients: clues from a biomarker study.

Authors:  Francesco D Tiziano; Rosa Lomastro; Lorena Di Pietro; Maria Barbara Pasanisi; Stefania Fiori; Carla Angelozzi; Emanuela Abiusi; Corrado Angelini; Gianni Sorarù; Alessandra Gaiani; Tiziana Mongini; Liliana Vercelli; Gessica Vasco; Giuseppe Vita; Gian Luca Vita; Sonia Messina; Luisa Politano; Luigia Passamano; Grazia Di Gregorio; Cristina Montomoli; Chiara Orsi; Angela Campanella; Renato Mantegazza; Lucia Morandi
Journal:  Eur J Hum Genet       Date:  2012-10-17       Impact factor: 4.246

4.  A natural history study of late onset spinal muscular atrophy types 3b and 4.

Authors:  S Piepers; L H van den Berg; F Brugman; H Scheffer; M Ruiterkamp-Versteeg; B G van Engelen; C G Faber; M de Visser; W-L van der Pol; J H J Wokke
Journal:  J Neurol       Date:  2008-06-30       Impact factor: 4.849

Review 5.  Spinal muscular atrophy.

Authors:  Maryam Oskoui; Petra Kaufmann
Journal:  Neurotherapeutics       Date:  2008-10       Impact factor: 7.620

Review 6.  Biomarkers in rare disorders: the experience with spinal muscular atrophy.

Authors:  Francesco D Tiziano; Giovanni Neri; Christina Brahe
Journal:  Int J Mol Sci       Date:  2010-12-24       Impact factor: 5.923

7.  Exploratory evaluation of an eye-tracking system in patients with advanced spinal muscular atrophy type I receiving nusinersen.

Authors:  Yukako Yae; Kotaro Yuge; Toshiyuki Maeda; Fumio Ichinose; Muneaki Matsuo; Osamu Kobayashi; Kazuo Okanari; Yusei Baba; Chihiro Yonee; Shinsuke Maruyama; Minoru Shibata; Tatsuya Fujii; Madoka Chinen; Yushiro Yamashita
Journal:  Front Neurol       Date:  2022-09-30       Impact factor: 4.086
  7 in total

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