Results 11 to 20 of about 2,012 (138)

Health economic evaluation of risdiplam in patients with spinal muscular atrophy [PDF]

Фармакоэкономика, 2021
Background. Spinal muscular atrophies (SMA) are clinically and genetically heterogenous inherited orphan diseases leading to progressive spinal motoneurons degeneration and loss of function.
А. S. Kolbin, А. А. Kurylev, Yu. Е. Balykina, М. А. Proskurin, S. А. Mishinova   +4 more
doaj   +3 more sources

Protocol Adherence in Spinal Muscular Atrophy Treated with Nusinersen and Risdiplam: A Retrospective Registry-Based Pharmacoclinical Audit

Recent Trends in Pharmacology
Objective: Disease-modifying therapies such as nusinersen and risdiplam have significantly changed the management of spinal muscular atrophy (SMA). In Spain, both are reimbursed under a national pharmacoclinical protocol with registry-based monitoring ...
Roberto Lozano, Carina Bona
doaj   +2 more sources

A Convenient, Pd-Free Approach to the Synthesis of Risdiplam

Molecules
Several approaches to the synthesis of risdiplam, a pharmacologically relevant pyridopyrimidinone derivative, have been recently reported. However, most of these routes rely exclusively on palladium-catalyzed, cross-coupling reactions and involve low ...
Georgiy Korenev, Alexey A. Gutenev, Fyodor V. Antipin, Vladimir V. Chernyshov, Julia A. Shulgina, Maria P. Korobkina, Maxim B. Nawrozkij, Roman A. Ivanov   +7 more
doaj   +2 more sources

Cost‐utility analysis of nusinersen–risdiplam switch in patients with spinal muscular atrophy in Croatia: A discrete event simulation model

British Journal of Clinical Pharmacology, EarlyView.
Introduction In recent years, the treatment of spinal muscular atrophy (SMA), a rare disease, has significantly progressed, improving patients' survival and overall quality of life. However, current SMA treatments are expensive, and some (nusinersen) are very inconvenient for patients.
Andrej Belančić, Ivana Stević, Elvira Meni Maria Gkrinia, Dinko Vitezić, Slobodan Janković   +4 more
wiley   +2 more sources

A real-world, multicentre, epidemiological study in Czech and Slovak adults with spinal muscular atrophy treated with risdiplam [PDF]

Scientific Reports
Real-world evidence about risdiplam therapy in adults with 5q spinal muscular atrophy (SMA) remains limited to outcomes in small cohorts, heterogeneous endpoints, and a short follow-up.
Olesja Parmova, Krystof Prasil, Lenka Mokra, Michal Svoboda, Petr Ridzon, Jana Junkerova, Lucia Bakosova, Katarina Okalova, Lenka Jurikova, Miriam Kolnikova, Jana Haberlova   +10 more
doaj   +2 more sources

Myostatin/Smad3 in Spinal Muscular Atrophy: Biomarkers of Disease Severity and Treatment Response. [PDF]

Ann Clin Transl Neurol
ABSTRACT Objective Given the role of Myostatin (MSTN) in muscle homeostasis and its modulation by current spinal muscular atrophy (SMA) therapies, we investigated serum MSTN and, for the first time, its downstream effector Smad3 as candidate non‐invasive biomarkers in SMA patients undergoing disease‐modifying therapies (DMTs).
Riolo G, Dosi C, Parravicini S, Zanin R, Cheli M, Grigoli EG, Canciello A, Andreetta F, Masson R, Bonanno S, Marcuzzo S, Maggi L.   +11 more
europepmc   +2 more sources

Real-world pharmacoclinical implementation of risdiplam under a national SMA protocol: A hospital pharmacy registry-based case series

Pakistan Journal of Pharmaceutical Sciences
Background: Spinal muscular atrophy (SMA) is a rare neuromuscular disorder treated with disease-modifying therapies such as risdiplam. In Spain, its use is regulated by a national pharmacoclinical protocol that requires structured monitoring. Objectives:
Roberto Lozano, Aragn Health Service, Zaragoza, Spain   +3 more
doaj   +2 more sources

Assessment of safety and efficacy of risdiplam treatment in adults with spinal muscular atrophy [PDF]

Frontiers in Neurology
IntroductionRisdiplam has been shown to be safe, well tolerated, and improves or stabilizes motor function in individuals with SMA, but limited published data exists for adults.
Andrea Jaworek, Kathryn Jira, Matti Allen, Songzhu Zhao, Kristina Kelly, Trevor Moravec, Marco Tellez, Sarah Heintzman, Jerold Reynolds, Gary Sterling, Stephen J. Kolb, William David Arnold, Bakri Elsheikh   +12 more
doaj   +2 more sources

Risdiplam treatment has not led to retinal toxicity in patients with spinal muscular atrophy [PDF]

, 2021
Objective: Evaluation of ophthalmologic safety with focus on retinal safety in patients with spinal muscular atrophy (SMA) treated with risdiplam (EVRYSDI), a survival of motor neuron 2 splicing modifier associated with retinal toxicity in monkeys ...
Nora Denk, Emmanuel Barreau, Nave, S, Santarsiero, Diletta, Amorelli, GM, Yashiro, Shigeko, Furst-Recktenwald, S, Mercuri, E, Jacobsen, Bjoern, Giovanni Baranello, Tremolada, Gemma, Marzoli, SB, Jaber, Birgit, Barreau, Emmanuel, Denk, Nora, Nave, Stephane, Gorni, K, Sabine Fürst‐Recktenwald, Birgit Jaber, Gerber, Marianne, Stefania B. Marzoli, Sergott, RC, Baranello, Giovanni, Kane, S, Jaber, B, Robert C. Sergott, Stephane Nave, Mueller, Lutz, Scalco, RS, Lorenzo Orazi, Melissa SantaMaria, Mueller, L, Agnieszka Waskowska, Mercuri, Eugenio, Shigeko Yashiro, Gorni, Ksenija, Yashiro, S, Steven Kane, Orazi, L, Denk, N, Beres, Shannon, Amorelli, Giulia, Gemma Tremolada, SantaMaria, M, Gerber, M, Beres, S, Kane, Steven, Santarsiero, D, Waskowska, Agnieszka, Baranello, G, Orazi, Lorenzo, Fürst‐recktenwald, Sabine, Waskowska, A, Jacobsen, B, Santamaria, Melissa, Renata S. Scalco, Diletta Santarsiero, Shannon Beres, Marianne Gerber, Lutz Mueller, Bjoern Jacobsen, Tremolada, G, Giulia M. Amorelli, Ksenija Gorni, Marzoli, Stefania, Scalco, Renata, Sergott, Robert C., Barreau, E, Eugenio Mercuri   +68 more
core   +2 more sources

Risdiplam therapy in adults with 5q-SMA: observational study on motor function and treatment satisfaction

BMC Neurology
Background We aimed to describe the experience of a single neuromuscular center in Germany in treating adult spinal muscular atrophy (SMA) patients with risdiplam and to analyze motor function and treatment satisfaction during a follow-up period up to 20 
Bogdan Bjelica, Camilla Wohnrade, Iraima Cespedes, Alma Osmanovic, Olivia Schreiber-Katz, Susanne Petri   +5 more
doaj   +2 more sources