Invitation to Roche ’s virtual event on 24-month data of Evrysdi in patients with type 2 or type 3 spinal muscular atrophy presented at the 2021 MDA virtual conference

We are pleased to invite investors and analysts to participate in our virtual event on Friday, 19 March, 2021, highlighting Roche data presented during the 2021 Muscular Dystrophy Association - MDA -Virtual Clinical and Scientific Conference, from 15th to 18th February.
Source: Roche Investor Update - Category: Pharmaceuticals Source Type: news

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The objective is to compare the disease course of untreated and Spinraza-treated patients with spinal muscular atrophy (SMA) as assessed by continuous measure issued from a wearable device (ActiMyo Ⓡ). ActiMyoⓇ, a magneto-inertial device, permits gait and activity analysis of patients in an uncontrolled environment. The European Medical Agency qualified the 95th centile of stride velocity (SV95C), a digital outcome computed through the sensors, as a secondary endpoint in Duchenne muscular dystrophy (DMD).
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research
This study analyses the relationships between hand strength and function in ambulant and non-ambulant patients with Duchenne muscular dystrophy (DMD) or spinal muscular atrophy (SMA). The maximal handgrip and key pinch strength were measured with the MyoGrip and MyoPinch dynamometers, respectively. Hand function was assessed using the MoviPlate, the Motor Function Measure items for distal upper limb (MFM D3 UL) and the Cochin scale. The data of 91 DMD and 77 SMA patients from 3 natural history studies were analysed (age range 6-31 years).
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research
Spinal muscular atrophy (SMA) is a rare neurodegenerative disease characterized by progressive muscular weakness. The management of SMA is resource intensive, with patients largely dependent on caregivers through their lives. The economic and social costs of SMA for patients and caregivers have been demonstrated in studies in Europe and the US. The primary objective of this study was to characterize the burden of SMA in Canada. Data was collected via online surveys distributed through two patient groups (CureSMA Canada and Muscular Dystrophy Canada), including demographics, clinical characteristics, healthcare resource use...
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research
Onuf’s nucleus is a small group of neurons located in the ventral horns of the sacral spinal cord. The motor neurons (MNs) of Onuf’s nucleus innervate striated voluntary muscles of the pelvic floor and are histologically and biochemically comparable to the other somatic spinal MNs. However, curiously, these neurons also show some autonomic-like features as, for instance, they receive a strong peptidergic innervation. The review provides an overview of the histological, biochemical, metabolic, and gene expression peculiarities of Onuf’s nucleus. Moreover, it describes the aging-related pathologies as well ...
Source: Frontiers in Neuroanatomy - Category: Neurology Source Type: research
This review highlights recent advances in gene-specific therapies for neuromuscular diseases of childhood, including spinal muscular atrophy, Duchenne muscular dystrophy, and centronuclear myopathy.Seminars in Neurology
Source: Medscape Today Headlines - Category: Consumer Health News Tags: Neurology & Neurosurgery Journal Article Source Type: news
on CL Abstract Spinal Muscular Atrophy (SMA) is an autosomal recessive neuromuscular disease caused by deletions or mutations in the survival motor neuron (SMN1) gene. An important hallmark of disease progression is the pathology of neuromuscular junctions (NMJs). Affected NMJs in the SMA context exhibit delayed maturation, impaired synaptic transmission, and loss of contact between motor neurons and skeletal muscle. Protection and maintenance of NMJs remains a focal point of therapeutic strategies to treat SMA, and the recent implication of the NMJ-organizer Agrin in SMA pathology suggests additional NMJ organizi...
Source: Biochemical and Biophysical Research communications - Category: Biochemistry Authors: Tags: Biochem Biophys Res Commun Source Type: research
a U Abstract Spinal muscular atrophy and muscular dystrophy Duchenne belong to the group of rare neuromuscular diseases manifesting in early childhood. Therapeutic options for some of these rare monogenic diseases have changed significantly in recent years. Molecular therapies such as direct gene transfer or alternative processing of the disease-specific gene play an important role in this transformation.In particular, the course of 5q-associated spinal muscle atrophy has changed significantly due to the availability of such causal therapies, while the results of ongoing studies are still pending for most muscle d...
Source: Bundesgesundheitsblatt, Gesundheitsforschung, Gesundheitsschutz - Category: International Medicine & Public Health Authors: Tags: Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz Source Type: research
Background: Drooling related to bulbar weakness and dysfunction is a common concern in patients with neuromuscular disease. While there are numerous medications to manage sialorrhea, they are often limited by side effects and lack of efficacy. Botulinum toxin has shown to benefit ALS patients in a few studies, but there is scant data on the benefit in other neuromuscular conditions.Objective: To assess the effectiveness of Botulinum toxin in reducing sialorrhea in patients with various neuromuscular disease.Design/Methods: 25 patients (19M, 6F; 54.36 ± 17.09 yr) with documented neuromuscular illness and concern for ...
Source: Frontiers in Neurology - Category: Neurology Source Type: research
From 30th September to 1st October 2019 a workshop was organized on sitting for persons with neuromuscular disorders, specifically focusing on Duchenne muscular dystrophy (DMD), Spinal muscular atrophy (SMA), congenital muscular dystrophy (CMD) and myopathy (CM), in Musholm Ferie-Sport-Konference in Kors ør, Denmark. Recruitment for occupational therapists was done in Western European countries; participating countries were Denmark, Norway, Sweden, Belgium, France and the Netherlands. A pre-workshop questionnaire was sent around concerning the use of standard evaluating methods and possible interve ntions.
Source: Neuromuscular Disorders - Category: Neurology Authors: Tags: Workshop report Source Type: research
From 30th September till 1st October 2019 a workshop was organized on sitting of persons with neuromuscular disorders, specifically focusing on Duchenne muscular dystrophy (DMD), Spinal muscular atrophy (SMA), congenital muscular dystrophy (CMD) and myopathy (CM), in Musholm Ferie-Sport- Konference in Kors ør, Denmark. Recruitment for occupational therapists was done in western European countries, participating countries were Denmark, Norway, Sweden, Belgium, France and the Netherlands. A pre-workshop questionnaire was send around concerning the use of standard evaluating methods and possible interve ntions.
Source: Neuromuscular Disorders - Category: Neurology Authors: Tags: Workshop report Source Type: research
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