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Antisense therapy is a form of treatment that uses antisense oligonucleotides (ASOs) to target messenger RNA (mRNA). ASOs are capable of altering mRNA expression through a variety of mechanisms, including ribonuclease H mediated decay of the pre-mRNA, direct steric blockage, and exon content modulation through splicing site binding on pre-mRNA. [1]
Golodirsen is one of the very few FDA-approved exon-skipping therapy for Duchenne muscular dystrophy, although the clinical benefits of the medication are yet to established. [ 1 ] [ 3 ] While the development of golodirsen needed huge financing, it is only applicable to a small subset of people with Duchenne muscular dystrophy.
The most common side effects include upper respiratory tract infections, cough, fever, headache, joint pain and throat pain. [ 2 ] [ 5 ] Casimersen was approved for medical use in the United States in February 2021, [ 1 ] [ 2 ] [ 6 ] and it is the first FDA-approved targeted treatment for people who have a confirmed mutation of the DMD gene ...
Gapmer-based therapeutics also have the potential for side effects. For example, Kynamro has been shown to induce injection site reactions, nausea, headaches, flu-like symptoms, and hepatotoxic reactions. [9] Side effects of Inotersen include thrombocytopenia, glomerulonephritis, injection site reactions, nausea, headache, fatigue, and fever [10]
The approval of aducanumab, despite the lack of evidence to support its therapeutic effects, led to controversy about the FDA’s approval process and a reluctance to prescribe the drug.
Tofersen, sold under the brand name Qalsody, is a medication used for the treatment of amyotrophic lateral sclerosis (ALS). [2] Tofersen is an antisense oligonucleotide that targets the production of superoxide dismutase 1, an enzyme whose mutant form is commonly associated with amyotrophic lateral sclerosis.
The US Food and Drug Administration (FDA) approved inotersen in October 2018. [6] The application for inotersen was granted orphan drug designation. [10] The FDA approved inotersen based on evidence from one clinical trial (Trial 1/NCT01737398) that included 172 participants with hereditary transthyretin-mediated amyloidosis. [6]
The most common side effects include injection site reactions. [3] Nedosiran was approved for medical use in the United States in September 2023. [1] [4] [5] [3] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [6]