Deepika vegiraju at 13:05, 19 August 2021

2021-08-19T13:05:46Z

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== '''What is Eteplirsen?''' ==

* Eteplirsen ('''EXONDYS 51''') is an '''antisense oligonucleotide''' used for the treatment of [[Duchenne muscular dystrophy]] (DMD).

[[File:Eteplirsen.svg|thumb]]

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== '''What are the uses of this medicine?''' ==

* This medicine is used for the treatment of [[Duchenne muscular dystrophy]] (DMD) in patients who have a '''confirmed mutation of the DMD gene''' that is amenable to '''exon 51 skipping'''.
* This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51.

== '''How does this medicine work?''' ==

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* Eteplirsen is designed to '''bind to exon 51 of dystrophin pre-mRNA''', resulting in '''exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping'''.
* Exon skipping is intended to '''allow for production of an internally truncated [[dystrophin]] protein''', which was evaluated in Study 2 and Study 3.

== '''Who Should Not Use this medicine ?''' ==

* This medicine have no usage limitations.

== '''What drug interactions can this medicine cause?''' ==

* No clinically important drug interactions have been observed with EXONDYS 51.

== '''Is this medicine FDA approved?''' ==

* It was approved for use in the United States in 2016.

== '''How should this medicine be used?''' ==

'''Recommended Dosage:'''
* The recommended dose of EXONDYS 51 is '''30 milligrams per kilogram '''administered once weekly as a 35 to 60 minute intravenous infusion via an in-line 0.2 micron filter.

'''Administration'''
* Dilution required prior to administration.
* Application of a topical anesthetic cream to the infusion site prior to administration of EXONDYS 51 may be considered.
* EXONDYS 51 is administered via intravenous infusion. Flush the intravenous access line with 0.9% Sodium Chloride Injection, USP, prior to and after infusion.
* Infuse the diluted EXONDYS 51 solution over 35 to 60 minutes via an in-line 0.2 micron filter. Do not mix other medications with EXONDYS 51 or infuse other medications concomitantly via the same intravenous access line.
* If a hypersensitivity reaction occurs, consider slowing the infusion or interrupting the EXONDYS 51 therapy
* If a dose of EXONDYS 51 is missed, it may be administered as soon as possible after the scheduled time.

== '''What are the dosage forms and brand names of this medicine?''' ==

This medicine is available in fallowing doasage form:
'''As Injection:'''
* 100 mg/2 mL (50 mg/mL) in single-dose vial
* 500 mg/10 mL (50 mg/mL) in single-dose vial

This medicine is available in fallowing brand namesː
*'''EXONDYS 51'''

=='''What side effects can this medication cause?'''==

The most common side effects of this medicine include:
* Balance disorder
* Vomiting
* Contact [[dermatitis]]

==''' What special precautions should I follow?''' ==

* [[Hypersensitivity]] reactions, including rash and [[urticaria]], [[pyrexia]], flushing, cough, [[dyspnea]], [[bronchospasm]], and [[hypotension]], have occurred in patients who were treated with EXONDYS 51. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy.

== '''What to do in case of emergency/overdose?''' ==

* There is no experience with overdose of EXONDYS 51.

== '''Can this medicine be used in pregnancy?''' ==

* There are no human or animal data available to assess the use of EXONDYS 51 during pregnancy.

=='''Can this medicine be used in children?'''==

* EXONDYS 51 is indicated for the treatment of [[Duchenne muscular dystrophy]] (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping, including pediatric patients.

== '''What are the active and inactive ingredients in this medicine?''' ==

'''Active ingredient:'''
* eteplirsen

'''Inactive ingredients:'''
* sodium chloride
* potassium chloride
* potassium phosphate, monobasic
* sodium phosphate, dibasic, anhydrous
* sodium hydroxide
* hydrochloric acid
* water

== '''Who manufactures and distributes this medicine?''' ==

* '''Packager:''' [[Sarepta Therapeutics]], Inc.

== '''What should I know about storage and disposal of this medication?''' ==

* Store EXONDYS 51 at 2°C to 8°C (36°F to 46°F).
* Do not freeze.
* Protect from light and store EXONDYS 51 in the original carton until ready for use.

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{{Other drugs for disorders of the musculo-skeletal system}}
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[[Category:Antisense RNA]]
[[Category:Muscular dystrophy]]
[[Category:Therapeutic gene modulation]]

Eteplirsen - Revision history

Deepika vegiraju at 13:05, 19 August 2021