Eosinophilic Oesophagitis in Spinal Muscular Atrophy
Though rare, spinal muscular atrophy (SMA) does sometimes co-occur with other rare conditions. Both SMA type 2 and eosinophilic oesophagitis (EoE) occur in approximately 0.16-4 per 10,000 people,1–4 but the conditions have also been simultaneously observed in...
Alternate Nusinersen Administration Strategies
In the pivotal clinical trials that support FDA approval, nusinersen was administered via intrathecal injection. However, this approach requires that patients receive multiple intrathecal injections separated in time by weeks. During each infusion, the clinician must...
Spinal Muscular Atrophy: Electromyography
Spinal Muscular Atrophy (SMA) is a progressive neuromuscular disease associated with typically proximal muscle weakness and atrophy due to degeneration of the anterior horn cells of the spinal cord.1 Prior to genetic testing as the gold standard diagnostic method for...
Spinal Muscular Atrophy Type 3 Subtypes
Spinal Muscular Atrophy (SMA) type 3, also known as Kugelberg Welander disease1, is diagnosed in fewer than 20% of patients with childhood-onset SMA.2 Patients with SMA type 3 typically will experience an insidious onset of leg weakness after they achieve independent...
Nusinersen
Nusinersen (Spinraza) is the first FDA-approved, disease-modifying therapy for the treatment of spinal muscular atrophy (SMA). Nusinersen is an antisense oligonucleotide that binds to a sequence downstream of exon 7 in the SMN2 gene. The oligonucleotide blocks the...HCP — SMA News Today
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