Atesidorsen
Atesidorsen
Atesidorsen is an investigational antisense oligonucleotide (ASO) designed for the treatment of certain genetic disorders. It is a synthetic strand of nucleic acid that binds to specific mRNA sequences, modulating gene expression. Atesidorsen is primarily being studied for its potential to treat diseases caused by genetic mutations that lead to the production of abnormal proteins.
Mechanism of Action[edit | edit source]
Atesidorsen works by targeting and binding to specific mRNA sequences in cells. This binding can alter the splicing of pre-mRNA, degrade the mRNA through RNase H-mediated cleavage, or block the translation of the mRNA into protein. By modulating the expression of genes, atesidorsen can potentially reduce the production of harmful proteins or restore the production of functional proteins in genetic disorders.
Clinical Applications[edit | edit source]
Atesidorsen is being investigated for its use in treating a variety of genetic disorders, including:
- Duchenne Muscular Dystrophy (DMD): Atesidorsen may be used to skip specific exons in the dystrophin gene, allowing for the production of a truncated but functional dystrophin protein.
- Spinal Muscular Atrophy (SMA): By modulating the splicing of the SMN2 gene, atesidorsen can increase the production of the survival motor neuron (SMN) protein.
Development and Trials[edit | edit source]
Atesidorsen is currently in various stages of clinical trials. These trials are designed to assess its safety, efficacy, and optimal dosing in patients with specific genetic mutations. The outcomes of these trials will determine the potential approval and use of atesidorsen in clinical practice.
Safety and Side Effects[edit | edit source]
As with any investigational drug, atesidorsen may have side effects. Common side effects observed in clinical trials include injection site reactions, flu-like symptoms, and elevated liver enzymes. Long-term safety data is still being collected.
Regulatory Status[edit | edit source]
Atesidorsen has not yet been approved by major regulatory agencies such as the FDA or EMA. It is available only through clinical trials or expanded access programs.
Also see[edit | edit source]
 | This pharmacology-related article is a stub. You can help WikiMD by expanding it. |
Search WikiMD
Ad.Tired of being Overweight? Try W8MD's physician weight loss program.
Semaglutide (Ozempic / Wegovy and Tirzepatide (Mounjaro / Zepbound) available.
Advertise on WikiMD
|
WikiMD's Wellness Encyclopedia |
| Let Food Be Thy Medicine Medicine Thy Food - Hippocrates |
Translate this page: - East Asian
中文,
日本,
한국어,
South Asian
हिन्दी,
தமிழ்,
తెలుగు,
Urdu,
ಕನ್ನಡ,
Southeast Asian
Indonesian,
Vietnamese,
Thai,
မြန်မာဘာသာ,
বাংলা
European
español,
Deutsch,
français,
Greek,
português do Brasil,
polski,
română,
русский,
Nederlands,
norsk,
svenska,
suomi,
Italian
Middle Eastern & African
عربى,
Turkish,
Persian,
Hebrew,
Afrikaans,
isiZulu,
Kiswahili,
Other
Bulgarian,
Hungarian,
Czech,
Swedish,
മലയാളം,
मराठी,
ਪੰਜਾਬੀ,
ગુજરાતી,
Portuguese,
Ukrainian
Medical Disclaimer: WikiMD is not a substitute for professional medical advice. The information on WikiMD is provided as an information resource only, may be incorrect, outdated or misleading, and is not to be used or relied on for any diagnostic or treatment purposes. Please consult your health care provider before making any healthcare decisions or for guidance about a specific medical condition. WikiMD expressly disclaims responsibility, and shall have no liability, for any damages, loss, injury, or liability whatsoever suffered as a result of your reliance on the information contained in this site. By visiting this site you agree to the foregoing terms and conditions, which may from time to time be changed or supplemented by WikiMD. If you do not agree to the foregoing terms and conditions, you should not enter or use this site. See full disclaimer.
Credits:Most images are courtesy of Wikimedia commons, and templates Wikipedia, licensed under CC BY SA or similar.
Contributors: Prab R. Tumpati, MD
