Pages that link to "Q33869164"
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The following pages link to Prevalence, incidence and carrier frequency of 5q-linked spinal muscular atrophy - a literature review. (Q33869164):
Displaying 48 items.
- Indirect estimation of the prevalence of spinal muscular atrophy Type I, II, and III in the United States (Q47144354) (← links)
- Motor Neuron Gene Therapy: Lessons from Spinal Muscular Atrophy for Amyotrophic Lateral Sclerosis (Q47152662) (← links)
- Overview of Current Drugs and Molecules in Development for Spinal Muscular Atrophy Therapy (Q48144714) (← links)
- Advances in therapy for spinal muscular atrophy: promises and challenges. (Q50052600) (← links)
- Inflammation in CNS Neurodegenerative Diseases. (Q51765219) (← links)
- An overview of the Cure SMA membership database: Highlights of key demographic and clinical characteristics of SMA members. (Q52609730) (← links)
- Nusinersen for spinal muscular atrophy. (Q54974497) (← links)
- Antisense oligonucleotides in neurological disorders. (Q55018458) (← links)
- Position Statement: Sharing of Clinical Research Data in Spinal Muscular Atrophy to Accelerate Research and Improve Outcomes for Patients. (Q55258215) (← links)
- Treatment Algorithm for Infants Diagnosed with Spinal Muscular Atrophy through Newborn Screening. (Q55265441) (← links)
- A Phase 1 healthy male volunteer single escalating dose study of the pharmacokinetics and pharmacodynamics of risdiplam (RG7916, RO7034067), a SMN2 splicing modifier (Q57282958) (← links)
- Global, regional, and national burden of motor neuron diseases 1990-2016: a systematic analysis for the Global Burden of Disease Study 2016 (Q58617336) (← links)
- Global burden of motor neuron diseases: mind the gaps (Q58617338) (← links)
- UBA1/GARS-dependent pathways drive sensory-motor connectivity defects in spinal muscular atrophy (Q58698132) (← links)
- Spinal muscular atrophy within Amish and Mennonite populations: Ancestral haplotypes and natural history (Q58762172) (← links)
- Efficacy and Safety of Valproic Acid for Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis (Q63090193) (← links)
- Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients (Q64062294) (← links)
- Humanising the mouse genome piece by piece (Q64097689) (← links)
- High Healthcare Resource Use in Hospitalized Patients with a Diagnosis of Spinal Muscular Atrophy Type 1 (SMA1): Retrospective Analysis of the Kids' Inpatient Database (KID). (Q64954166) (← links)
- Gene therapies for axonal neuropathies: Available strategies, successes to date, and what to target next (Q89452029) (← links)
- The analysis of the association between the copy numbers of survival motor neuron gene 2 and neuronal apoptosis inhibitory protein genes and the clinical phenotypes in 40 patients with spinal muscular atrophy: Observational study (Q89501954) (← links)
- Carrier Frequency of Spinal Muscular Atrophy in a Large-scale Korean Population (Q89750567) (← links)
- Carrier frequency of spinal muscular atrophy in Thailand (Q90026254) (← links)
- Palliative Care in SMA Type 1: A Prospective Multicenter French Study Based on Parents' Reports (Q90056837) (← links)
- Direct Medical Costs of Spinal Muscular Atrophy in the Catalonia Region: A Population-Based Analysis (Q90122747) (← links)
- Fetal Gene Therapy Using a Single Injection of Recombinant AAV9 Rescued SMA Phenotype in Mice (Q90205161) (← links)
- Health, wellbeing and lived experiences of adults with SMA: a scoping systematic review (Q90275352) (← links)
- Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy (Q90478065) (← links)
- Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment (Q90555235) (← links)
- One Year of Newborn Screening for SMA - Results of a German Pilot Project (Q90593635) (← links)
- The implementation of newborn screening for spinal muscular atrophy: the Australian experience (Q90679196) (← links)
- Muscle regulates mTOR dependent axonal local translation in motor neurons via CTRP3 secretion: implications for a neuromuscular disorder, spinal muscular atrophy (Q90728074) (← links)
- Presentation and Management Patterns of Lower Urinary Tract Symptoms in Adults Due to Rare Inherited Neuromuscular Diseases (Q90798872) (← links)
- Physical exercise training for type 3 spinal muscular atrophy (Q92033286) (← links)
- Genomic analysis of a spinal muscular atrophy (SMA) discordant family identifies a novel mutation in TLL2, an activator of growth differentiation factor 8 (myostatin): a case report (Q92330801) (← links)
- Survival, Motor Function, and Motor Milestones: Comparison of AVXS-101 Relative to Nusinersen for the Treatment of Infants with Spinal Muscular Atrophy Type 1 (Q92416237) (← links)
- Healthcare utilisation in children with SMA type 1 treated with nusinersen: a single centre retrospective review (Q92465463) (← links)
- Newborn Screening for Spinal Muscular Atrophy in China Using DNA Mass Spectrometry (Q92539534) (← links)
- Management and current status of spinal muscular atrophy: a retrospective multicentre claims database analysis (Q92559996) (← links)
- De-duplicating patient records from three independent data sources reveals the incidence of rare neuromuscular disorders in Germany (Q92993542) (← links)
- Circulating MyomiRs as Potential Biomarkers to Monitor Response to Nusinersen in Pediatric SMA Patients (Q93006923) (← links)
- Implementation of population-based newborn screening reveals low incidence of spinal muscular atrophy (Q94957207) (← links)
- Experiences of caregivers of children with spinal muscular atrophy participating in the expanded access program for nusinersen: a longitudinal qualitative study (Q98163592) (← links)
- NGS-based spinal muscular atrophy carrier screening of 10,585 diverse couples in China: a pan-ethnic study (Q98945157) (← links)
- Maximum bite force in patients with spinal muscular atrophy during the first year of nusinersen therapy - A pilot study (Q99240515) (← links)
- Intrathecal Access Through Suboccipital Port in Patients With Spinal Muscular Atrophy and Complex Spines: Case Series and Technical Note (Q99240881) (← links)
- CMAP changes upon symptom onset and during treatment in spinal muscular atrophy patients: lessons learned from newborn screening (Q100504061) (← links)
- Current Clinical Applications of in vivo Gene Therapy with AAVs (Q104289829) (← links)