Results 81 to 90 of about 2,012 (138)

Managing Spinal Muscular Atrophy: A Look at the Biology and Treatment Strategies

Biology
Since its discovery in the late 19th century, spinal muscular atrophy (SMA) has had a significant medical and societal impact, primarily affecting newborns, toddlers, and young adults.
Arianna Vezzoli, Daniele Bottai, Raffaella Adami   +2 more
doaj   +1 more source

Diving into progress: a review on current therapeutic advancements in spinal muscular atrophy

Frontiers in Neurology
Spinal muscular atrophy (SMA) is an uncommon disorder associated with genes characterized by the gradual weakening and deterioration of muscles, often leading to substantial disability and premature mortality.
Pankaj Bagga, Sudhakar Singh, Gobind Ram, Subham Kapil, Avtar Singh   +4 more
doaj   +1 more source

Comparative efficacy of risdiplam and nusinersen in Type 2 and 3 spinal muscular atrophy patients: A cohort study using real-world data

Background: Three medications have been approved for spinal muscular atrophy (SMA) treatment. No head-to-head clinical trials have directly compared the efficacy of nusinersen and risdiplam.
Hosseini, S. A., Ghaletaki, G. Z., Montazerlotfelahi, H., Abdi, A., Toosi, M. B., Nafissi, S., Tavasoli, A., Nazari, S. S. H., Bakhtiary, H., Khajeh, A., Taghdiri, M. M., Inaloo, S., Nikbakht, S., Jafari, N., Fatehi, F., Heidari, M., Memarian, S., Hosseiny, S. M. M., Akbari, M. G., Badv, R. S., Abbaskhanian, A., Shariatmadari, F., Momen, A., Barzegar, M., Aminzadeh, V., Babaei, M., Heshmat, R., Ziaadini, B., Mollamohammadi, M., Babaee, M., Ghazavi, M., Ashrafi, M. R., Farshadmoghadam, H., Nasehi, M. M.   +33 more
core   +1 more source

Serum circulating cell-free messenger RNA profile response to risdiplam treatment in adult patients with late-onset spinal muscular atrophy. [PDF]

Brain Commun
Leo M, Schmitt LI, Liebig KC, Hezel S, Neuhoff S, Roos A, Ruck T, Kleinschnitz C, Hagenacker T.   +8 more
europepmc   +1 more source

Generative Chemistry Platform for Small Molecules Targeting RNA: A Case Study for Chemical Optimization. [PDF]

Comput Struct Biotechnol J
Allen TEH, Boyd SM, Bonnet M, Khan RT.
europepmc   +1 more source

Real-world 12-month outcomes of Risdiplam in spinal muscular atrophy types 2 and 3: A Brazilian cohort. [PDF]

Clinics (Sao Paulo)
Camelo CG, Mendonça RH, Moreno CAM, Polido GJ, Carvalho EV, Teixeira WCDS, Hayashi DS, Brito JJDC, Matsui Junior C, Zanoteli E.   +9 more
europepmc   +1 more source

Spinal muscular atrophy: Biology, pathogenesis, and therapeutic advances. [PDF]

Ther Adv Neurol Disord
René CA, Parks RJ.
europepmc   +1 more source

Ceftriaxone-Resistant <i>Escherichia coli</i> Sepsis in an Infant With Spinal Muscular Atrophy Type 1 Receiving Risdiplam: A Case Report. [PDF]

Clin Case Rep
Zetawi M, Shoman R, Afana A, Aldarak S, Draidi R, AbuBaha B, Jaradat R, Khouli F, Salameh H.   +8 more
europepmc   +1 more source

Spinal muscular atrophy in the disease-modifying therapy era: successes, limitations and future directions. [PDF]

Front Mol Med
Sexton MM, Crow EW, Peng CA.
europepmc   +1 more source

Motor function score changes in severe 5q spinal muscular atrophy during risdiplam treatment: an observational longitudinal nationwide cohort study. [PDF]

EClinicalMedicine
Vermeer LM, Asselman FL, van Eijk RPA, Cuppen I, van der Woude DR, Visser-de Heus BMHEA, Hogervorst SMJ, Ruyten TJ, Bartels B, Wadman RI, van der Pol WL.   +10 more
europepmc   +1 more source