* Casimersen AMONDYS 45 is an antisense [[oligonucleotide]] indicated for the treatment of [[Duchenne muscular dystrophy]] (DMD).
[[File:Casimersen.svg|thumb|right|Chemical structure of Casimersen]]
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'''Casimersen''' is a medication used in the treatment of [[Duchenne muscular dystrophy]] (DMD), a genetic disorder characterized by progressive muscle degeneration and weakness. Casimersen is specifically designed for patients with a confirmed mutation of the DMD gene that is amenable to exon 45 skipping.
* This medicine 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 45 skipping.
Casimersen is an [[antisense oligonucleotide]] that targets exon 45 of the [[dystrophin]] gene. By binding to this specific exon, Casimersen induces exon skipping during [[mRNA]] processing, which allows for the production of a truncated but functional dystrophin protein. This mechanism aims to restore the production of dystrophin, which is deficient in patients with Duchenne muscular dystrophy.
* This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with AMONDYS 45.
== '''How does this medicine work?''' ==
== Administration ==
* Casimersen is designed to '''bind to exon 45 of dystrophin pre-mRNA resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 45 skipping'''.
Casimersen is administered via intravenous infusion. The dosing regimen is typically once weekly, and the dosage is based on the patient's weight. The treatment is usually continued as long as the patient shows clinical benefit and does not experience unacceptable side effects.
* Exon 45 skipping is intended to allow for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 45 skipping.
== '''Who Should Not Use this medicine ?''' ==
== Side Effects ==
* This medicine have no usage limitation
Common side effects of Casimersen include:
* [[Headache]]
* [[Fever]]
* [[Cough]]
* [[Upper respiratory tract infection]]
== '''What drug interactions can this medicine cause?''' ==
Serious side effects may include [[kidney toxicity]], and regular monitoring of kidney function is recommended during treatment.
== Clinical Trials ==
== '''Is this medicine FDA approved?''' ==
Casimersen has undergone clinical trials to evaluate its efficacy and safety in patients with Duchenne muscular dystrophy. These trials have demonstrated that Casimersen can increase dystrophin production in muscle tissue, which is associated with improved clinical outcomes in some patients.
* It was approved for use in the United States in 2021.
== Related Pages ==
== '''How should this medicine be used?''' ==
* [[Duchenne muscular dystrophy]]
* [[Antisense oligonucleotide]]
* [[Exon skipping]]
* [[Dystrophin]]
* Serum [[cystatin C]], urine dipstick, and urine protein-to-creatinine ratio (UPCR) should be measured before starting AMONDYS 45.
[[Category:Muscular dystrophy treatments]]
* Consider measurement of [[glomerular filtration rate]] prior to initiation of AMONDYS 45.
[[Category:Antisense therapy]]
* Monitoring for kidney toxicity during treatment is recommended.
* Obtain the urine sample prior to infusion of AMONDYS 45 or at least 48 hours after infusion.
'''Recommended Dosage'''
* The recommended dosage of AMONDYS 45 is''' 30 milligrams per kilogram '''administered once weekly as a 35 to 60-minute intravenous infusion via an in-line 0.2 micron filter.
* If a dose of AMONDYS 45 is missed, it may be administered as soon as possible after the scheduled dose.
'''Administration'''
* Application of a topical anesthetic cream to the infusion site prior to administration of AMONDYS 45 may be considered.
* AMONDYS 45 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 AMONDYS 45 over 35 to 60 minutes via an in-line 0.2 micron filter.
* Do not mix other medication with AMONDYS 45 or infuse other medications concomitantly via the same intravenous access with AMONDYS 45.
== '''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 in a single-dose vial
This medicine is available in fallowing brand namesː
*'''AMONDYS'''
=='''What side effects can this medication cause?'''==
The most common side effects of this medicine include:
* upper respiratory tract infection
* cough
* [[pyrexia]]
* headache
* [[arthralgia]]
* [[oropharyngeal pain]]
==''' What special precautions should I follow?''' ==
* This medicine may cause kidney toxicity.
* Kidney function should be monitored; [[creatinine]] may not be a reliable measure of renal function in DMD patients.
== '''What to do in case of emergency/overdose?''' ==
* {{overdose}}
== '''Can this medicine be used in pregnancy?''' ==
* There are no human or animal data available to assess the use of AMONDYS 45 during pregnancy.
=='''Can this medicine be used in children?'''==
* AMONDYS 45 is indicated for the treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping, including pediatric patients.
== '''What should I know about storage and disposal of this medication?''' ==
* Store AMONDYS 45 at 2°C to 8°C (36°F to 46°F). Do not freeze. Store in original carton until ready for use to protect from light.
{{Other drugs for disorders of the musculo-skeletal system}}
Casimersen is a medication used in the treatment of Duchenne muscular dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration and weakness. Casimersen is specifically designed for patients with a confirmed mutation of the DMD gene that is amenable to exon 45 skipping.
Casimersen is an antisense oligonucleotide that targets exon 45 of the dystrophin gene. By binding to this specific exon, Casimersen induces exon skipping during mRNA processing, which allows for the production of a truncated but functional dystrophin protein. This mechanism aims to restore the production of dystrophin, which is deficient in patients with Duchenne muscular dystrophy.
Casimersen is administered via intravenous infusion. The dosing regimen is typically once weekly, and the dosage is based on the patient's weight. The treatment is usually continued as long as the patient shows clinical benefit and does not experience unacceptable side effects.
Casimersen has undergone clinical trials to evaluate its efficacy and safety in patients with Duchenne muscular dystrophy. These trials have demonstrated that Casimersen can increase dystrophin production in muscle tissue, which is associated with improved clinical outcomes in some patients.
