نتائج البحث - "Ashish Dugar"

1

دورية أكاديمية

Patient engagement in clinical trial design for rare neuromuscular disorders: impact on the DELIVER and ACHIEVE clinical trials

المؤلفون: Patricia Furlong, Ashish Dugar, Molly White

المصدر: Research Involvement and Engagement, Vol 10, Iss 1, Pp 1-11 (2024)

مصطلحات موضوعية: Patient-focused drug development, Patient and public involvement, Duchenne muscular dystrophy, Myotonic dystrophy, Medicine, Medicine (General), R5-920

وصف الملف: electronic resource

العلاقة: https://doaj.org/toc/2056-7529Test

الوصول الحر: https://doaj.org/article/15b21f5fbf914c78a217489a730162f0Test

View record in DOAJ

النص الكامل

2

Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial

المؤلفون: Ashish Dugar, Eugenio Mercuri, Xiaodong Wang, Volker Straub, Baoguang Han, Daniela Leone, Navid Khan, Dan Wang, Erica Koenig, Laurent Servais, Andreea Mihaela Seferian, Michela Guglieri, Mariacristina Scoto, Francesco Muntoni

المساهمون: University of Oxford, Institut de Myologie, Commissariat à l'énergie atomique et aux énergies alternatives (CEA)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Association française contre les myopathies (AFM-Téléthon)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Sorbonne Université (SU)-Centre National de la Recherche Scientifique (CNRS), Centre Hospitalier Universitaire de Liège (CHU-Liège), Université de Liège, Università cattolica del Sacro Cuore = Catholic University of the Sacred Heart [Roma] (Unicatt), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Newcastle University [Newcastle], Newcastle Upon Tyne Hospitals NHS Foundation Trust, University College of London [London] (UCL), Great Ormond Street Hospital for Children [London] (GOSH), Sarepta Therapeutics, University of Oxford [Oxford], I-Motion Institut [CHU Trousseau], Commissariat à l'énergie atomique et aux énergies alternatives (CEA)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Association française contre les myopathies (AFM-Téléthon)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Sorbonne Université (SU)-Centre National de la Recherche Scientifique (CNRS)-Commissariat à l'énergie atomique et aux énergies alternatives (CEA)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Association française contre les myopathies (AFM-Téléthon)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Sorbonne Université (SU)-Centre National de la Recherche Scientifique (CNRS), Università cattolica del Sacro Cuore [Roma] (Unicatt), Fondazione 'Policlinico Universitario A. Gemelli' [Rome], Gestionnaire, Hal Sorbonne Université

المصدر: Nucleic Acid Therapeutics
Nucleic Acid Therapeutics, In press, ⟨10.1089/nat.2021.0043⟩
Nucleic Acid Therapeutics, Mary Ann Liebert, Inc. publishers, In press, ⟨10.1089/nat.2021.0043⟩

مصطلحات موضوعية: Duchenne muscular dystrophy, medicine.medical_specialty, [SDV.NEU.NB]Life Sciences [q-bio]/Neurons and Cognition [q-bio.NC]/Neurobiology, Population, Oligonucleotides, Walk Test, golodirsen, Biochemistry, Dystrophin, 03 medical and health sciences, FEV1/FVC ratio, 0302 clinical medicine, Internal medicine, Multicenter trial, Drug Discovery, Genetics, medicine, Clinical endpoint, Humans, education, Molecular Biology, 030304 developmental biology, 0303 health sciences, education.field_of_study, business.industry, [SDV.NEU.NB] Life Sciences [q-bio]/Neurons and Cognition [q-bio.NC]/Neurobiology, Exons, medicine.disease, Original Papers, Exon skipping, 3. Good health, Clinical trial, Muscular Dystrophy, Duchenne, [SDV.SP.PHARMA] Life Sciences [q-bio]/Pharmaceutical sciences/Pharmacology, Ambulatory, [SDV.SP.PHARMA]Life Sciences [q-bio]/Pharmaceutical sciences/Pharmacology, Molecular Medicine, business, 030217 neurology & neurosurgery, exon skipping