Pages that link to "Q33885457"
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The following pages link to A multi-source approach to determine SMA incidence and research ready population (Q33885457):
Displaying 18 items.
- Natural History, Trial Readiness and Gene Discovery: Advances in Patient Registries for Neuromuscular Disease. (Q45951421) (← links)
- Motor Neuron Gene Therapy: Lessons from Spinal Muscular Atrophy for Amyotrophic Lateral Sclerosis (Q47152662) (← links)
- An overview of the Cure SMA membership database: Highlights of key demographic and clinical characteristics of SMA members. (Q52609730) (← links)
- Spinal muscular atrophy type I and the dual role of viruses: An interview with Professor Basil T. Darras, Professor of Neurology (Pediatrics) at Harvard Medical School. (Q52647744) (← links)
- Position Statement: Sharing of Clinical Research Data in Spinal Muscular Atrophy to Accelerate Research and Improve Outcomes for Patients. (Q55258215) (← links)
- Spinal muscular atrophy within Amish and Mennonite populations: Ancestral haplotypes and natural history (Q58762172) (← links)
- SMArtCARE - A platform to collect real-life outcome data of patients with spinal muscular atrophy (Q61446053) (← links)
- "Getting ready for the adult world": how adults with spinal muscular atrophy perceive and experience healthcare, transition and well-being (Q64086577) (← links)
- Gene therapies for axonal neuropathies: Available strategies, successes to date, and what to target next (Q89452029) (← links)
- RESTORE: A Prospective Multinational Registry of Patients with Genetically Confirmed Spinal Muscular Atrophy - Rationale and Study Design (Q89626564) (← links)
- Carrier frequency of spinal muscular atrophy in Thailand (Q90026254) (← links)
- Health, wellbeing and lived experiences of adults with SMA: a scoping systematic review (Q90275352) (← links)
- High-throughput genetic newborn screening for spinal muscular atrophy by rapid nucleic acid extraction from dried blood spots and 384-well qPCR (Q92297063) (← links)
- Newborn Screening for Spinal Muscular Atrophy in China Using DNA Mass Spectrometry (Q92539534) (← links)
- De-duplicating patient records from three independent data sources reveals the incidence of rare neuromuscular disorders in Germany (Q92993542) (← links)
- A national spinal muscular atrophy registry for real world evidence (Q96129213) (← links)
- The ties that bind: functional clusters in limb-girdle muscular dystrophy (Q98163745) (← links)
- Molecular diagnosis and genetic counseling for spinal muscular atrophy (SMA) (Q104567667) (← links)