Zobrazeno 1 - 10
of 44
pro vyhledávání: '"Kristin J Krosschell"'
Autor:
Jean Fitzpatrick Timmerberg, Kristin J Krosschell, Sally Dunaway Young, David Uher, Chris Yun, Jacqueline Montes
Publikováno v:
PLoS ONE, Vol 16, Iss 4, p e0249279 (2021)
Background and purposeWith the availability and development of disease-modifying therapies for individuals with spinal muscular atrophy (SMA), new emerging phenotypes must be characterized, and potential new treatment paradigms tested. There is an ur
Externí odkaz:
https://doaj.org/article/78a96d9acb0941d99d5b7151582019a1
Autor:
John T Kissel, Charles B Scott, Sandra P Reyna, Thomas O Crawford, Louise R Simard, Kristin J Krosschell, Gyula Acsadi, Bakri Elsheik, Mary K Schroth, Guy D'Anjou, Bernard LaSalle, Thomas W Prior, Susan Sorenson, Jo Anne Maczulski, Mark B Bromberg, Gary M Chan, Kathryn J Swoboda, Project Cure Spinal Muscular Atrophy Investigators' Network
Publikováno v:
PLoS ONE, Vol 6, Iss 7, p e21296 (2011)
BACKGROUNDMultiple lines of evidence have suggested that valproic acid (VPA) might benefit patients with spinal muscular atrophy (SMA). The SMA CARNIVAL TRIAL was a two part prospective trial to evaluate oral VPA and L-carnitine in SMA children. Part
Externí odkaz:
https://doaj.org/article/141aa541d70f4d909428010ea6f22fd6
Autor:
Kathryn J Swoboda, Charles B Scott, Thomas O Crawford, Louise R Simard, Sandra P Reyna, Kristin J Krosschell, Gyula Acsadi, Bakri Elsheik, Mary K Schroth, Guy D'Anjou, Bernard LaSalle, Thomas W Prior, Susan L Sorenson, Jo Anne Maczulski, Mark B Bromberg, Gary M Chan, John T Kissel, Project Cure Spinal Muscular Atrophy Investigators Network
Publikováno v:
PLoS ONE, Vol 5, Iss 8, p e12140 (2010)
Valproic acid (VPA) has demonstrated potential as a therapeutic candidate for spinal muscular atrophy (SMA) in vitro and in vivo.Two cohorts of subjects were enrolled in the SMA CARNIVAL TRIAL, a non-ambulatory group of "sitters" (cohort 1) and an am
Externí odkaz:
https://doaj.org/article/2a5f3943a1fe48acb839ca7db24528ca
Autor:
Kathryn J Swoboda, Charles B Scott, Sandra P Reyna, Thomas W Prior, Bernard LaSalle, Susan L Sorenson, Janine Wood, Gyula Acsadi, Thomas O Crawford, John T Kissel, Kristin J Krosschell, Guy D'Anjou, Mark B Bromberg, Mary K Schroth, Gary M Chan, Bakri Elsheikh, Louise R Simard
Publikováno v:
PLoS ONE, Vol 4, Iss 5, p e5268 (2009)
UNLABELLED:Preliminary in vitro and in vivo studies with valproic acid (VPA) in cell lines and patients with spinal muscular atrophy (SMA) demonstrate increased expression of SMN, supporting the possibility of therapeutic benefit. We performed an ope
Externí odkaz:
https://doaj.org/article/cf5b116376a74737bee79e5d14d205d2
Autor:
Kristin J. Krosschell, Laurey Brown, Katie Hoffman, Katelyn B. Zumpf, Hannah Munson, Jessa Bidwell, Duncan P. Schulte, Abigail N. Schwaede, Amber N. Buehner, Christine J. DiDonato, Nancy L. Kuntz, Vamshi K. Rao
Publikováno v:
Journal of Neuromuscular Diseases. 10:337-348
Background: Ambulatory individuals with spinal muscular atrophy experience weakness and impairments of speed and endurance. This leads to decreased motor skill performance required for daily living including transitioning from floor to stand, climbin
Autor:
Katelyn Zumpf, Kristin J. Krosschell, Michael Kiefer, Elise L. Townsend, Sarah D Simeone, Kathryn J. Swoboda, Leah J. Welty
Publikováno v:
Neuromuscul Disord
As trials and treatments for Spinal Muscular Atrophy (SMA) rapidly evolve, understanding the natural history and potential utility of the 10-meter walk/run test (10MWRT) in ambulant individuals is critical. Study aims were to: 1) establish change ove
Autor:
Kristin J Krosschell, Sally Dunaway Young, Ilse Peterson, Mary Curry, Allison Mazzella, Jill Jarecki, Rosángel Cruz
Publikováno v:
Physical Therapy. 102
Disease-modifying therapies for spinal muscular atrophy (SMA) are rapidly changing the outlook for many individuals by substantially altering the clinical course, phenotypic expression, and functional outcomes. Physical therapists have played critica
Publikováno v:
Neuromuscular Disorders. 31:397-408
This study describes the current landscape of physical therapy practice recommendations in the United States for children with spinal muscular atrophy (SMA) and their relationship to current SMA standard of care (SMA-SOC) guidelines. Pediatric physic
Publikováno v:
Orphanet Journal of Rare Diseases
Orphanet Journal of Rare Diseases, Vol 17, Iss 1, Pp 1-7 (2022)
Orphanet Journal of Rare Diseases, Vol 17, Iss 1, Pp 1-7 (2022)
Recent advances in technology and expanding therapeutic opportunities in neuromuscular disorders has resulted in greater interest in and development of remote assessments. Over the past year, the rapid and abrupt COVID-19 shutdowns and stay-at-home o
Autor:
Tina Duong, Kristin J. Krosschell, Meredith K. James, Leslie Nelson, Lindsay N. Alfano, Katy Eichinger, Elena Mazzone, Kristy Rose, Linda P. Lowes, Anna Mayhew, Julaine Florence, Wendy King, Claudia R. Senesac, Michelle Eagle
Publikováno v:
Frontiers in Genetics, Vol 12 (2021)
Frontiers in Genetics
Frontiers in Genetics
Critical components of successful evaluation of clinical outcome assessments (COAs) in multisite clinical trials and clinical practice are standardized training, administration, and documented reliability of scoring. Experiences of evaluators, alongs