Results 121 to 130 of about 2,700 (197)

Review of cardiac safety in onasemnogene abeparvovec gene therapy: translation from preclinical to clinical findings [PDF]

, 2022
Deepa H. Chand, Rui Sun, Karim Diab, Damien Kenny, Francis Funyuy Tukov   +4 more
openalex   +1 more source

Clinical decision making around commercial use of gene and genetic therapies for spinal muscular atrophy

Neurotherapeutics
Spinal muscular atrophy is no longer a leading cause of inherited infant death in the United States. Since 2016, three genetic therapies have been approved for the treatment of spinal muscular atrophy.
Megan A. Waldrop
doaj   +1 more source

Reduction in Perioperative Risk in Patients with Spinal Muscular Atrophy Following the Release of Disease-Modifying Therapies: An Analysis of the National Surgical Quality Improvement Program Database

Children
Background/Objectives: Spinal muscular atrophy (SMA) is a progressive neurodegenerative disease resulting in proximal muscle weakness and paralysis. SMA treatment has radically changed in the past 10 years thanks to the development of novel therapies ...
Erin Toaz, Nisha Pinto, Keith Kilner, Eric Cheon   +3 more
doaj   +1 more source

Outcomes of Early-Treated Infants with Spinal Muscular Atrophy: A Multicenter, Retrospective Cohort Study [PDF]

Introduction/aims: While prompt identification and treatment of infants with spinal muscular atrophy (SMA) can ameliorate outcomes, variability persists. This study assessed management and outcomes of early-treated infants with SMA. Methods: We analyzed
Arya, Kapil, Brandsema, John F., Farah, Hiba, Farrar, Michelle A., Felker, Marcia V., Fisher, Mark, Gibbons, Melissa, Goedeker, Natalie L., Hamid, Omer Abdul, Harmelink, Matthew, Herbert, Karen, Kichula, Elizabeth, King, Kiana, Kuntz, Nancy L., Lakhotia, Arpita, Lee, Bo Hoon, Parsons, Julie, Rehborg, Rebecca, Rogers, Amanda, Veerapaniyan, Aravindhan, Zaidman, Craig M.   +20 more
core   +2 more sources

CO41 Real-World Outcomes of Nusinersen or Onasemnogene Abeparvovec (OA) Monotherapy, or Switching to OA from Nusinersen in SMA Patients Aged ≥6 Months [PDF]

, 2023
Omar Dabbous, Mei Yang, Mihaela Georgieva, W. Toro, Nicole LaMarca, Anish Patel, Anna Anderson, Hanane Akbarnejad, Sandra P. Reyna   +8 more
openalex   +1 more source

Clinicogenetic characterization and response to disease-modifying therapies in spinal muscular atrophy: real-world experience from a reference center in Southern Brazil

Jornal de Pediatria
Objective: Spinal Muscular Atrophy linked to chromosome 5q (SMA) is an autosomal recessive neurodegenerative disease characterized by progressive proximal muscle atrophy and weakness.
Ana Letícia Amorim de Albuquerque, Júlia Kersting Chadanowicz, Isabela Possebon Bevilacqua, Ana Lucia Portella Staub, Pablo Brea Winckler, Patricia Zambone da Silva, Simone Chaves Fagondes, Renata Salatti Ferrari, Claudia Denise de Oliveira Trojahn, Viviane Zechlinski Sacharuk, Thayne Woycinck Kowalski, Karina Carvalho Donis, Michele Michelin Becker, Jonas Alex Morales Saute   +13 more
doaj   +1 more source

Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series [PDF]

, 2020
Deepa H. Chand, Craig M. Zaidman, Kapil Arya, Rachel Millner, Michelle A. Farrar, Fiona Mackie, Natalie L. Goedeker, Vikas R. Dharnidharka, Raja Dandamudi, Sandra P. Reyna   +9 more
openalex   +1 more source

Neuromuscular diseases: genomics-driven advances

Genomics & Informatics
Neuromuscular diseases (NMDs) are a group of rare disorders characterized by significant genetic and clinical complexity. Advances in genomics have revolutionized both the diagnosis and treatment of NMDs. While fewer than 30 NMDs had known genetic causes
Anna Cho
doaj   +1 more source

Gene therapy: A Revolution in Medicine or a Risky Game with our DNA? [PDF]

Introduction: The aim of this review is to provide an overview of the applications of gene therapies in various diseases and to highlight areas where further research is needed.
Długozima, Patrycja, Grywińska, Weronika, Grzeszczuk, Paulina, Głowacka, Aleksandra, Kalisz, Agnieszka, Kozakiewicz, Julia, Kościelecki, Kamil, Mączewska, Klaudia, Okońska, Aleksandra, Skorulska, Iwona   +9 more
core   +1 more source

Therapeutic Efficacy and Safety of onasemnogene abeparvovec Gene Therapy for Spinal Muscular Atrophy Type 1: Updated Systematic Review with Meta-Analysis [PDF]

Sarah Mohamed Alfaqaih, George Hanen, Hazem E. Mohammed, Mohamed Nasser, Mohamed E. Haseeb, Hatem Yaser, Shehab Yaser, Mohamed Khalafalla Darwish   +7 more
openalex   +1 more source