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Combination therapy with onasemnogene and risdiplam in spinal muscular atrophy type 1.
Oechsel, Kelly F; Cartwright, Michael S.
  • Oechsel KF; Department of Neurology, Wake Forest School of Medicine, Winston-Salem, North Carolina, USA.
  • Cartwright MS; Department of Neurology, Wake Forest School of Medicine, Winston-Salem, North Carolina, USA.
Muscle Nerve ; 64(4): 487-490, 2021 10.
Article in English | MEDLINE | ID: covidwho-1318732
ABSTRACT
INTRODUCTION/

AIMS:

There are currently three medications approved for spinal muscular atrophy (SMA), but the use of these medications in combination has not been well described.

METHODS:

This is a retrospective report of four cases of SMA treated with dual onasemnogene and risdiplam therapy at our institution.

RESULTS:

Following onasemnogene therapy, all four patients experienced a perceived plateau of therapeutic benefit, at which time daily risdiplam was started. Transient fatigue and weakness was seen in two patients following risdiplam initiation, but this resolved within 1 mo. One patient was hospitalized with severe acute respiratory syndrome coronavirus-2 (SARS-CoV-2) and post-viral pneumonia, weeks following risdiplam initiation. No other adverse effects related to onasemnogene and risdiplam combination therapy were identified and all patients experienced objective and subjective improvement.

DISCUSSION:

Combination therapy with onasemnogene and risdiplam in patients with SMA appears to be well-tolerated. Further large prospective trials are needed to determine whether dual therapy is more efficacious than monotherapy, and to identify rare adverse events that may occur with the use of combination therapy.
Subject(s)
Keywords

Full text: Available Collection: International databases Database: MEDLINE Main subject: Pyrimidines / Azo Compounds / Biological Products / Recombinant Fusion Proteins / Spinal Muscular Atrophies of Childhood Type of study: Case report / Diagnostic study / Observational study / Prognostic study Limits: Female / Humans / Infant / Male Language: English Journal: Muscle Nerve Year: 2021 Document Type: Article Affiliation country: Mus.27375

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Full text: Available Collection: International databases Database: MEDLINE Main subject: Pyrimidines / Azo Compounds / Biological Products / Recombinant Fusion Proteins / Spinal Muscular Atrophies of Childhood Type of study: Case report / Diagnostic study / Observational study / Prognostic study Limits: Female / Humans / Infant / Male Language: English Journal: Muscle Nerve Year: 2021 Document Type: Article Affiliation country: Mus.27375