Unrestricted oral feedings reported by caregivers and affected persons across severity of SMA
Maureen Lefton‐Greif talks about a study showing that unrestricted oral feeding – measured using a modified version of the FOIS scale – is more common in adults with less severe types of spinal muscular atrophy, and that some loss of function occurs over time.
Let’s Talk… SMA at EPNS 2022 | Francesco Danilo Tiziano
We speak to Francesco Danilo Tiziano about newborn screening for spinal muscular atrophy, including his experience in Italy and the impact of screening on patient outcomes.
Let’s Talk… SMA at EPNS 2022 | Mariacristina Scoto
Mariacristina Scoto talks about real-world studies on disease-modifying treatments for spinal muscular atrophy, and how some of the data presented at EPNS 2022 could impact clinical practice.
Let’s Talk… SMA at EPNS 2022 | Laurent Servais
Laurent Servais talks about the challenges in treatment decision making for spinal muscular atrophy and how they could be addressed, drawing on early-phase anti-myostatin antibody research as a potential treatment solution if shown to be beneficial in phase 3 trials.
Pooled analysis demonstrates favorable safety profile for risdiplam
Maria Mazurkiewicz-Bełdzińska outlines a pooled analysis of data from the FIREFISH, SUNFISH, and JEWELFISH trials, which demonstrates that risdiplam has a favorable benefit–risk profile in patients with spinal muscular atrophy (3:42).
Feasibility of assisted 6-minute cycle test shown in non-ambulatory SMA patients
Tina Duong talks through a real-world follow-up study demonstrating that treatment with nusinersen is associated with continued stabilization of motor function over 2–3 years among adult patients with spinal muscular atrophy.
Nusinersen treatment may improve aerobic capacity in adult SMA patients
Jacqueline Montes discusses the results of a post-hoc analysis demonstrating a significant improvement in aerobic capacity among adult patients with spinal muscular atrophy treated with nusinersen (3:03).
Treatment linked to continued stabilization of motor function in adults with SMA
Tina Duong talks through a real-world follow-up study demonstrating that treatment with nusinersen is associated with continued stabilization of motor function over 2–3 years among adult patients with spinal muscular atrophy.
Study identifies miRNAs upregulated in SMA
Emanuela Abiusi outlines the results of a study showing that certain microRNAs are upregulated in muscle biopsy and serum samples taken from people with spinal muscular atrophy, and discusses the potential of these microRNAs as a target for SMN-independent therapies (4:46).
EAN 2020 | Teriflunomide shows benefits in children with relapsing forms of MS
Tanuja Chitnis tells us about the TERIKIDS study showing a numerical reduction in clinical relapse risk and a significant reduction in time to clinical relapse with teriflunomide in paediatric patients with relapsing-remitting multiple sclerosis (5:29).
