Last Updated: 09/26/2026
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Found 6 publications
Natural history of limb girdle muscular dystrophy R1 (LGMDR1): a GRASP consortium study.
Journal: Neuromuscular disorders : NMD
Published: December 10, 2025
Clinical Trial Readiness in Limb Girdle Muscular Dystrophy R1 (LGMDR1): A GRASP Consortium Study.
Journal: Annals of clinical and translational neurology
Published: January 08, 2025
Practical Guidance for the Use of Patisiran in the Management of Polyneuropathy in Hereditary Transthyretin-Mediated Amyloidosis.
Journal: Therapeutics and clinical risk management
Published: August 05, 2023
Advance Care Planning Among Patients With Amyotrophic Lateral Sclerosis: Patient Perspectives on Goals of Care Conversations.
Journal: The American journal of hospice & palliative care
Published: May 02, 2023
Treatment preference among patients with spinal muscular atrophy (SMA): a discrete choice experiment.
Journal: Orphanet journal of rare diseases
Published: August 04, 2020
Early experiences of nusinersen for the treatment of spinal muscular atrophy: Results from a large survey of patients and caregivers.
Journal: Muscle & nerve
Published: March 31, 2020
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Last Updated: 09/26/2026