Golodirsen definition

Golodirsen means [**].
Sample 1

Examples of Golodirsen in a sentence

  • Golodirsen is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer (PMO) subclass.” Highlights of Prescribing Information (Dec.

  • Claimspaid in error by ineligibility under COBRA will be reviewed for collection.

  • Golodirsen is excreted mostly unchanged in the urine; therefore, renal dysfunction increases golodirsen exposure.

  • Recommendation 4: Vote to Prior Authorize Amondys 45™, Viltepso®, and Vyondys 53™The Drug Utilization Review Board recommends the prior authorization of Amondys 45™ (Casimersen), Viltepso® (Viltolarsen), and Vyondys 53™ (Golodirsen) with the following criteria:Amondys 45™ (Casimersen), Viltepso® (Viltolarsen), and Vyondys 53™ (Golodirsen) Approval Criteria:1.

  • Golodirsen (Vyondys 53, approved 2019) and viltolarsen (Viltepso, approved 2020) received biomarker‐based accelerated approvals for the treatment of a subset of DMD patients with mutations in the dystrophin gene that are amenable to exon 53 skipping.

  • Deflazacort, Eteplirsen, and Golodirsen for Duchenne Muscular Dystrophy: Effectiveness and Value.

  • A similar medication, Golodirsen (brand name Vyondys 53), was approved by the FDA December 2019, and can be used in patients who have adeletion in the dystrophin gene that is ame- nable to Exon 53 skipping.13 Boys with DMD are also treated with glucocorticoids, specifically prednisone or deflazacort (brand name Emflaza), to prolong ambulation, arm function, and cardiopulmonary function.

  • Mean fiber inten- sity analysis showed a significant increase from baseline in de novo dystrophin production (P<0.001) and confirmed dystro- phin sarcolemmal localization.Conclusions: Golodirsen is the second PMO shown to increase dystrophin expression and facilitate sarcolemmal locali- zation following initiation of exon skipping.

  • According to the clinical investigator, the pharmacy manual was not available until after the study had started, which may have contributed to these errors.These dosing errors, summarized in Table 2, were not included in the protocol deviation list for this NDA submission (please refer to summary of sponsor inspection summary below).Page 10 Clinical Inspection SummaryNDA #211970, Golodirsen 2.

  • Golodirsen is designed to bind to exon 53 of dystrophin pre-mRNA, resulting in exclusion, or “skipping,” of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping.

Related to Golodirsen

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