Results 81 to 90 of about 1,718 (175)

Development of a chromatographic method for the analysis of risdiplam in serum extracts

Bioanalysis
Risdiplam has been used to treat spinal muscular atrophy for 3 years. There are limited number of papers devoted to its analytics. Until now, risdiplam and its metabolites have only been analyzed using a C18 column, while the sample preparation method involved protein precipitation. Risdiplam was analyzed using reversed-phase UHPLC.
Balińska, Natalia, Lemska, Anna, Mazurkiewicz-Bełdzińska, Maria, Studzińska, Sylwia   +3 more
openaire   +3 more sources

Considerations for Treatment in Clinical Care of Spinal Muscular Atrophy Patients

Children
Spinal Muscular Atrophy is a neurodegenerative disease which can lead to muscle weakness, paralysis, and in some cases death. There are many factors that contribute to the severity of symptoms and those factors can be used to determine the best course of
Stephanie Voight, Kapil Arya
doaj   +1 more source

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

Clinical characteristics of the included cases treated with risdiplam and the associated adverse events.

Clinical characteristics of the included cases treated with risdiplam and the associated adverse events.
Limei Liu (134888), Lurong Yu (18086654)
core   +1 more source

Evaluating Therapeutic Outcomes in Spinal Muscular Atrophy: An Indian Experience

Annals of Indian Academy of Neurology
Background and Objectives: Spinal muscular atrophy (SMA) is a progressive neuromuscular disorder that leads to anterior horn cell loss. Recent advancements in disease-modifying therapies (DMTs) offer promising new treatment options.
Smilu Mohanlal, PM Mubeena, Hafsa Hussain, Manjula Anand, Satish Kumar, VV Ashraf, Tajimal A Rabia, Sujith Janardhanan, Suresh Kumar   +8 more
doaj   +1 more source

Longitudinal motor function and biomarker correlates in treated adult spinal muscular atrophy: a single-center cohort study

Frontiers in Neurology
BackgroundLong-term clinical trajectories and accessible biomarkers in adult spinal muscular atrophy (SMA) remain insufficiently characterized under real-world treatment conditions.
Chikashi Yano, Masahiro Ando, Akiko Yoshimura, Yujiro Higuchi, Yoshikatsu Noda, Jun-Hui Yuan, Tomonori Nakamura, Takahiro Hobara, Risa Nagatomo, Fumikazu Kojima, Mika Yuji, Yu Hiramatsu, Satoshi Nozuma, Akihiro Hashiguchi, Akihiro Hashiguchi, Yusuke Sakiyama, Hiroshi Takashima   +16 more
doaj   +1 more source

Risdiplam

American Journal of Health-System Pharmacy, 2020
openaire   +2 more sources

ID036 Judicialização da saúde para medicamentos órfãos: Redução dos custos com aquisição centralizada pelo Ministério da Saúde no caso do Risdiplam

Jornal de Assistência Farmacêutica e Farmacoeconomia
Introdução A judicialização da saúde é um fenômeno que tem impactado a gestão de recursos do Sistema Único de Saúde (SUS), especialmente com a judicialização no tratamento de doenças raras, como a Atrofia Muscular Espinhal (AME) que se utilizam de ...
Ângela Maria Bagattini, Ligia Oliveira Almeida Mendes   +1 more
doaj   +1 more source

Survival motor neuron protein is the optimal biomarker for evaluating the risdiplam treatment

Brain and Development
The clinical use of three disease-modifying drugs approved in Japan for the treatment of spinal muscle atrophy (SMA) has increased. Therefore, there is an increasing need for biomarkers to evaluate therapeutic interventions.Individuals were treated with risdiplam (25 patients, male:female =12:13) and evaluated before starting the medication and after 1,
Tamaki, Kato, Noriko, Otsuki, Mamoru, Yokomura, Naoko, Shima, Kota, Kajima, Moeka, Shioya, Hayato, Suzuki, Hidetaka, Wakabayshi, Kayoko, Saito   +8 more
openaire   +2 more sources