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

Autor: 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

Publikováno v: 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⟩

The aim of this Phase 1/2, 2-part, multicenter trial was to report clinical safety and efficacy of long-term golodirsen treatment among ambulatory patients with exon 53 skip-amenable Duchenne muscular dystrophy (DMD). Part 1 was a 12-week, randomized

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::84e23f53949f6934fac11e79b70202fb
http://europepmc.org/articles/PMC8817703

Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy

Autor: Kathleen Church, Samiah Al-Zaidy, Ashish Dugar, Kelly Lehman, Zarife Sahenk, Rachael A Potter, Sarah Lewis, Mark J. Hogan, Louise R. Rodino-Klapac, N. Miller, Carrie Nease, Richard Shell, Amanda Nicholl, Danielle A. Griffin, Jerry R. Mendell, M. Iammarino, Lindsay N. Alfano, Eric Pozsgai, Linda H. Cripe, Linda Lowes

Publikováno v: JAMA Neurol

Importance Micro-dystrophin gene transfer shows promise for treating patients with Duchenne muscular dystrophy (DMD) using recombinant adeno-associated virus serotype rh74 (rAAVrh74) and codon-optimized human micro-dystrophin driven by a skeletal and

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::86dd24b7ec12e659f034c13d0ad483f0
https://doi.org/10.1001/jamaneurol.2020.1484

Health Care Utilization and Costs in Patients with Generalized Anxiety Disorder Initiating Add-on Therapy with Benzodiazepines

Autor: Ariel Berger, Gerry Oster, Vamsi Bollu, Jose Alvir, John Edelsberg, Ashish Dugar, Ashish V. Joshi

Publikováno v: Health Outcomes Research in Medicine. 3:e45-e54

Objectives To examine patterns of health care utilization and costs in patients with generalized anxiety disorder (GAD) who begin treatment with benzodiazepine anxiolytics as add-on therapy. Study Design In a large US health insurance database, we id

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::0bbe5c0f748209be43fe06e5bc999f72
https://doi.org/10.1016/j.ehrm.2011.11.002

Progression of visual loss and time between initial assessment and treatment of wet age-related macular degeneration

Autor: Shaun Segal, Gaurav K. Shah, Sanjay Sharma, Jeffrey A. Bakal, Alejandro Oliver-Fernandez, Ashish Dugar

Publikováno v: Canadian Journal of Ophthalmology. 40:313-319

Purpose: To determine whether the time elapsed from initial (referral) diagnosis of neovascular (wet) age-related macular degeneration (AMD) to assessment and treatment by a retinal specialist is associated with visual deterioration in the intervenin

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0ca5f3fcf379ebdbf3cfcdc04ed864a9
https://doi.org/10.1016/s0008-4182(05)80074-2

Age-dependent loss of corticosterone modulation of central serotonin 5-HT1A receptor binding sites

Autor: Keck Bj, Lynn W. Maines, Ashish Dugar, Joan M. Lakoski

Publikováno v: Journal of Neuroscience Research. 53:86-98

A loss of endocrine and neurotransmitter system interactions, including corticosterone regulation of 5-HT1Areceptors, may underlie the age-related deficits in the hypothalamic-pituitary-adrenal (HPA) axis including adapting to stress. In this study,

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::8093d6038ecf8aa42fd9da5fe069fcf6
https://doi.org/10.1002/(sici)1097-4547(19980701)53:1<86::aid-jnr9>3.0.co