Results 41 to 50 of about 4,580 (188)
, 2022 Limited evidence exists on real-world adherence to nusinersen for the treatment of spinal muscular atrophy (SMA). Data are presented from a multi-site retrospective chart review of 86 adults with SMA initiating nusinersen at nine US centers between ...
Michelson, David +13 more
core +1 more source
Nationwide Epidemiology of Motor Neuron Diseases in Latvia (2020-2024): Incidence, Prevalence, and Clinical Characteristics. [PDF]
Eur J NeurolThis first nationwide study of motor neuron diseases (MNDs) in Latvia provides epidemiological and clinical data about 181 patients identified with motor neuron diseases during a 5‐year observational period revealing lower incidence and prevalence rates compared to other European countries.
Krutovs V +9 more
europepmc +2 more sources
Brain Sciences, 2021 The antisense oligonucleotide nusinersen has been shown to improve trunk and limb motor function in patients with spinal muscular atrophy (SMA). Bulbar dysfunction, which is regularly present in SMA, is not captured by standard motor scores, and ...
Svenja Brakemeier +7 more
doaj +1 more source
British Journal of Clinical Pharmacology, EarlyView.With multiple disease‐modifying therapies now available, treatment switching has become an important clinical consideration in the management of spinal muscular atrophy (SMA). While some switches are prompted by suboptimal clinical response, more commonly they are driven by treatment burden, convenience, or adverse events.
Andrej Belančić +4 more
wiley +1 more source
Orphanet Journal of Rare Diseases, 2023 Background As the first gene therapy for spinal muscular atrophy (SMA), nusinersen is supposed to be administrated via intrathecal injection regularly for a lifetime.
Zhen Wang +10 more
doaj +1 more source
Children, 2021 The purpose of this study was to explore early changes in patient and family caregiver report of quality of life and family impact during the transitional period of nusinersen use.
Meaghann S. Weaver +4 more
doaj +1 more source
British Journal of Clinical Pharmacology, EarlyView.Small and mid‐sized pharmaceutical innovators often have limited in‐house health economics and market access expertise, and may struggle to align development strategies of investigational medicinal products with health system needs and payer expectations.
Zoltán Kaló +5 more
wiley +1 more source
Increased chitotriosidase 1 concentration following nusinersen treatment in spinal muscular atrophy
Orphanet Journal of Rare Diseases, 2021 Background Studies regarding the impact of (neuro)inflammation and inflammatory response following repetitive, intrathecally administered antisense oligonucleotides (ASO) in 5q-associated spinal muscular atrophy (SMA) are sparse.
Maren Freigang +12 more
doaj +1 more source
Early‐stage health technology assessment of a curative gene therapy for multiple sclerosis
British Journal of Clinical Pharmacology, EarlyView.Aims Multiple sclerosis (MS) is associated with significant early morbidity, reduced life expectancy and substantial healthcare and societal costs. The primary objective of this study is to assess the early cost‐effectiveness potential of a novel gene therapy, IMMUTOL, for MS compared with current high‐efficacy treatment sequences.
Attila Imre, Balázs Nagy, Rok Hren
wiley +1 more source
Evolution of bulbar function in spinal muscular atrophy type 1 treated with nusinersen [PDF]
, 2022 Aim: To assess the evolution of bulbar function in nusinersen-treated spinal muscular atrophy type 1 (SMA1). Method: This single-centre retrospective study identified 24 patients (14 females and 10 males) with SMA1, treated with nusinersen between 2017 ...
Weststrate, Harriet +8 more
core +1 more source