Zobrazeno 1 - 10
of 142
pro vyhledávání: '"Eric A. Hoffman"'
Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy
Autor:
Paula R, Clemens, Vamshi K, Rao, Anne M, Connolly, Amy D, Harper, Jean K, Mah, Craig M, McDonald, Edward C, Smith, Craig M, Zaidman, Tomoyuki, Nakagawa, Eric P, Hoffman
Publikováno v:
Journal of neuromuscular diseases. 9(4)
Duchenne muscular dystrophy (DMD) is a rare, genetic disease caused by mutations in the DMD gene resulting in an absence of functional dystrophin protein. Viltolarsen, an exon 53 skipping therapy, has been shown to increase endogenous dystrophin leve
Autor:
Anabel S. De la Garza-Rodea, Steven A. Moore, Jesus Zamora-Pineda, Eric P. Hoffman, Karishma Mistry, Ashok Kumar, Jonathan B. Strober, Piming Zhao, Jung H. Suh, Julie D. Saba
Publikováno v:
International Journal of Molecular Sciences; Volume 23; Issue 14; Pages: 7579
International journal of molecular sciences, vol 23, iss 14
International journal of molecular sciences, vol 23, iss 14
Duchenne muscular dystrophy (DMD) is a congenital myopathy caused by mutations in the dystrophin gene. DMD pathology is marked by myositis, muscle fiber degeneration, and eventual muscle replacement by fibrosis and adipose tissue. Satellite cells (SC
Autor:
Eric P. Hoffman
Publikováno v:
The Febs Journal
Duchenne muscular dystrophy is the most common neuromuscular genetic disorder. This review describes the identification of the cause of the disorder in the late 1980s—dystrophin deficiency—and the emerging therapeutics enabled by increased unders
Autor:
Yusuke, Echigoya, Nhu, Trieu, William, Duddy, Hong M, Moulton, HaiFang, Yin, Terence A, Partridge, Eric P, Hoffman, Joe N, Kornegay, Frank A, Rohret, Christopher S, Rogers, Toshifumi, Yokota
Publikováno v:
International Journal of Molecular Sciences
Duchenne muscular dystrophy (DMD) is a lethal X-linked recessive disorder caused by mutations in the DMD gene and the subsequent lack of dystrophin protein. Recently, phosphorodiamidate morpholino oligomer (PMO)-antisense oligonucleotides (ASOs) targ
Autor:
Kanneboyina Nagaraju, Qi Long Lu, Melissa Morales, Yetrib Hathout, Umar Burki, James S. Novak, Aiping Zhang, Terence A. Partridge, Katie A. Edwards, Kathleen Tatum, Eric P. Hoffman, Margaret Benny Klimek, Jack H. Van der Mulen, James L Quinn, Alyson A. Fiorillo, John van den Anker, Volker Straub, Maria Candida Vila, Jessica F. Boehler
Publikováno v:
Journal of neuromuscular diseases. 8(s2)
Background: Phosphorodiamidate morpholino oligomer (PMO)-mediated exon skipping is currently used in clinical development to treat Duchenne muscular dystrophy (DMD), with four exon-skipping drugs achieving regulatory approval. Exon skipping elicits a
Autor:
Kanneboyina Nagaraju, Mansi V. Goswami, James S. Novak, Jesslyn S Zhang, Cinrg-Dnhs Investigators, Marissa L Barbieri, Eric P. Hoffman, Michael Olarewaju Ogundele, Yetrib Hathout, Utkarsh J. Dang
Publikováno v:
Life, Vol 11, Iss 827, p 827 (2021)
Life
Volume 11
Issue 8
Life
Volume 11
Issue 8
Duchenne muscular dystrophy (DMD) is a progressive muscle disease involving complex skeletal muscle pathogenesis. The pathogenesis is triggered by sarcolemma instability due to the lack of dystrophin protein expression, leading to Ca2+ influx, muscle
Publikováno v:
Journal of Neuromuscular Diseases
Duchenne muscular dystrophy (DMD) is a devastating, rare disease. While clinically described in the 19th century, the genetic foundation of DMD was not discovered until more than 100 years later. This genetic understanding opened the door to the deve
Autor:
Katie A. Edwards, James S. Novak, Kanneboyina Nagaraju, Qi Long Lu, Margaret Benny Klimek, Jessica F. Boehler, Bonnie Douglas, Yi-Wen Chen, Melissa Morales, Ning Li, Maria Candida Vila, Davi A. G. Mázala, Yetrib Hathout, John van den Anker, Eric P. Hoffman, Alyson A. Fiorillo, Alexander G. Fritz, Terence A. Partridge, Marshall W. Hogarth, Travis B. Kinder, Aiping Zhang
Publikováno v:
J Pathol
Exon skipping is a promising genetic therapeutic strategy for restoring dystrophin expression in the treatment of Duchenne muscular dystrophy (DMD). The potential for newly synthesized dystrophin to trigger an immune response in DMD patients, however
Autor:
Marisol Montolio, Nicole A. Datson, Francesco Muntoni, Eric P. Hoffman, Judith C.T. van Deutekom, Pallavi Lonkar, Michael Binks, Jane Owens, Virginia Arechavala-Gomeza, Glenn E. Morris, V. Ashutosh Rao, Rahul Phadke, Hendrik Neubert, Annemieke Aartsma-Rus, Jennifer E. Morgan
Publikováno v:
Journal of Neuromuscular Diseases
Representatives of academia, patient organisations, industry and the United States Food and Drug Administration attended a workshop on dystrophin quantification methodology. The aims of the workshop were to provide an overview of methods used to quan
Autor:
Yetrib Hathout, Utkarsh J. Dang, Eric P. Hoffman, Molly Barkhouse, Mamta Giri, Heather Gordish-Dressman, Kitipong Uaesoontrachoon, Michael Ziemba, Kanneboyina Nagaraju
Publikováno v:
PLoS ONE, Vol 16, Iss 2, p e0246507 (2021)
PLoS ONE
PLoS ONE
Duchenne muscular dystrophy is initiated by dystrophin deficiency, but downstream pathophysiological pathways such as membrane instability, NFĸB activation, mitochondrial dysfunction, and induction of TGFβ fibrosis pathways are thought to drive the