Duchenne expert physician perspectives on Duchenne newborn screening and early Duchenne care

por Armstrong, Niki, Schrader, Rachel, Fischer, Ryan, Crossnohere, Norah
Publicado 2022

Natural killer cells in liver transplantation: Can we harness the power of the immune checkpoint to promote tolerance?

por Halma, Jennifer, Pierce, Stephen, McLennan, Rebecca, Bradley, Todd, Fischer, Ryan
Publicado 2021

Diagnostic experiences of Duchenne families and their preferences for newborn screening: A mixed‐methods study

por Crossnohere, Norah L., Armstrong, Niki, Fischer, Ryan, Bridges, John F. P.
Publicado 2022

Neonatal Graves' Disease with Maternal Hypothyroidism

por Akangire, Gangaram, Cuna, Alain, Lachica, Charisse, Fischer, Ryan, Raman, Sripriya, Sampath, Venkatesh
Publicado 2017

Torsion of an Accessory Spleen in a Child With Biliary Atresia Splenic Malformation Syndrome

por Simon, David A., Fleishman, Nathan R., Choi, Pamala, Fraser, Jason D., Fischer, Ryan T.
Publicado 2020

Use and safety of prophylactic endoscopy from a single center serving urban and rural children with portal hypertension

por Slowik, Voytek, Bernardez, Anissa, Wasserkrug, Heather, Fischer, Ryan T., Daniel, James F., Grammatikopoulos, Tassos
Publicado 2022

A Comparison of Caregiver and Patient Preferences for Treating Duchenne Muscular Dystrophy

por Crossnohere, Norah L., Fischer, Ryan, Vroom, Elizabeth, Furlong, Patricia, Bridges, John F. P.
Publicado 2022

132 Caregivers Perspectives on Multidisciplinary Clinic Visits for Duchenne and Becker Muscular Dystrophy

por Fawcett, Kindann, Haynie, Cade, Roby, Tiffany, Wagner, Ellen, Schrader, Rachel, Fischer, Ryan, Veerapandiyan, Aravindhan
Publicado 2023

Unmet Therapeutic Needs of Non-Ambulatory Patients with Duchenne Muscular Dystrophy: A Mixed-Method Analysis

por Schuster, Anne L. R., Crossnohere, Norah L., Fischer, Ryan, Furlong, Patricia, Bridges, John F. P.
Publicado 2022

An electronic medical records-based approach to identify idiosyncratic drug-induced liver injury in children

por Sandritter, Tracy L., Goldman, Jennifer L., Habiger, Clayton J., Daniel, James F., Lowry, Jennifer, Fischer, Ryan T.
Publicado 2019

Readiness to Change and Prospective Effects of Weight Management Programs in Pediatric Nonalcoholic Fatty Liver Disease

por Slowik, Voytek, Wasserkrug, Heather, Fischer, Ryan T., Connelly, Mark, Deacy, Amanda D., Hampl, Sarah, Daniel, James F.
Publicado 2020

Development of a Clinical Global Impression of Change (CGI-C) and a Caregiver Global Impression of Change (CaGI-C) measure for ambulant individuals with Duchenne muscular dystrophy

por Staunton, Hannah, Trennery, Claire, Arbuckle, Rob, Guridi, Maitea, Zhuravleva, Elena, Furlong, Pat, Fischer, Ryan, Hall, Rebecca
Publicado 2021

A Mixed-Method Study Exploring Patient-Experienced and Caregiver-Reported Benefits and Side Effects of Corticosteroid Use in Duchenne Muscular Dystrophy

por Fischer, Ryan, Porter, Katherine, Donovan, Joanne M., Scavina, Mena T., Armstrong, Niki, Denger, Brian, Hasham, Shabir, Peay, Holly
Publicado 2023

The effect of neuropsychiatric medication on pediatric nonalcoholic fatty liver disease

por Ryan, Jamie L., Sherman, Ashley K., Heble, Daniel E., Friesen, Craig A., Daniel, James F., Fischer, Ryan T., Slowik, Voytek
Publicado 2022

Uric Acid as a Potential Peripheral Biomarker for Disease Features in Huntington’s Patients

por Corey-Bloom, Jody, Haque, Ameera, Aboufadel, Sameer, Snell, Chase, Fischer, Ryan S., Granger, Steven W., Granger, Douglas A., Thomas, Elizabeth A.
Publicado 2020

Levels of Interleukin-6 in Saliva, but Not Plasma, Correlate with Clinical Metrics in Huntington’s Disease Patients and Healthy Control Subjects

por Corey-Bloom, Jody, Fischer, Ryan S., Kim, Aeri, Snell, Chase, Parkin, Georgia M., Granger, Douglas A., Granger, Steven W., Thomas, Elizabeth A.
Publicado 2020

Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy: A qualitative preference study of patients and parents

por Landrum Peay, Holly, Fischer, Ryan, Tzeng, Janice P., Hesterlee, Sharon E., Morris, Carl, Strong Martin, Amy, Rensch, Colin, Smith, Edward, Ricotti, Valeria, Beaverson, Katherine, Wand, Hannah, Mansfield, Carol
Publicado 2019

How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration

por Furlong, Pat, Bridges, John F. P., Charnas, Lawrence, Fallon, Justin R., Fischer, Ryan, Flanigan, Kevin M., Franson, Timothy R., Gulati, Neera, McDonald, Craig, Peay, Holly, Sweeney, H. Lee
Publicado 2015

Patients’ and caregivers’ maximum acceptable risk of death for non‐curative gene therapy to treat Duchenne muscular dystrophy

por Peay, Holly L., Fischer, Ryan, Mange, Brennan, Paquin, Ryan S., Smith, Edward C., Sadosky, Alesia, Russo, Leo, Ricotti, Valeria, Rensch, Colin, Morris, Carl, Martin, Amy Strong, Ganot, Annie, Beaverson, Katherine, Mansfield, Carol
Publicado 2021

Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best–worst scaling experiment in caregivers and adult patients

por Paquin, Ryan S., Fischer, Ryan, Mansfield, Carol, Mange, Brennan, Beaverson, Katherine, Ganot, Annie, Martin, Amy Strong, Morris, Carl, Rensch, Colin, Ricotti, Valeria, Russo, Leo J., Sadosky, Alesia, Smith, Edward C., Peay, Holly L.
Publicado 2019