Question 1

What is GOLODIRSEN used for?

Accepted Answer

GOLODIRSEN (brand name Vyondys 53) is a Other indicated for See full prescribing information.

Question 2

How does GOLODIRSEN work?

Accepted Answer

GOLODIRSEN works through the following mechanism: 12.1 Mechanism of Action Golodirsen is designed to bind to exon 53 of dystrophin pre-mRNA resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping. Exon 53 skipping is intended to allow for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 53 skipping [see Clinical Studies ( 14 )]..

Question 3

What are the most common side effects of GOLODIRSEN?

Accepted Answer

Reported side effects vary by individual. The side-effects section on this page lists the events most frequently reported to FDA FAERS for GOLODIRSEN. FAERS is voluntary reporting, so it reflects what was reported, not absolute incidence.

Question 4

Is GOLODIRSEN the same as Vyondys 53?

Accepted Answer

Vyondys 53 is a brand name for GOLODIRSEN. They share the same active ingredient and FDA-approved indications. Biosimilars and authorised generics may differ in inactive ingredients such as buffers or preservatives.

GOLODIRSEN

Mechanism of Action

Indications

FDA Warnings

Known Side Effects

Community Discussions

Primary sources

Compare With

Formulations