TO TREAT DUCHENNE MUSCULAR DYSTROPHY WITH AGAMREE (VAMOROLONE)

  • Unique Paper ID: 180932
  • Volume: 12
  • Issue: 1
  • PageNo: 3442-3458
  • Abstract:
  • The dystrophin protein, which is necessary for safeguarding muscle cells during contraction, is not produced in Duchenne due to mutations in the DMD gene. Patients experience increasing muscular degeneration and atrophy as a result. Over time, chronic inflammation plays a role in the development of this damage. For individuals with Duchenne muscular dystrophy (DMD) who are two years of age or older, the dissociative steroid agamree (vamorolone) is used to maintain muscle function and reduce inflammation. Santhera Pharmaceuticals sold the rights to market Agamree in North America to Catalyst Pharmaceuticals. The treatment was first created by ReveraGen BioPharma. Current clinical management guidelines state that mechanical ventilatory assistance is typically started at night to address hypoventilation and sleep-related breathing issues sometime in the second to third decade of a patient's life. All patients eventually require help breathing, even throughout the day, to survive as their respiratory muscles continue to degenerate. Because of the disease's morbidity and mortality, DMD is linked to a significant burden on afflicted individuals, unpaid caregivers, and society at large. Mainly, the DMD is caused by a genetic mutation in the DMD gene causes dystrophin, a protein necessary for muscle function, to be absent, leading to progressive muscle weakness and degeneration. This is the cause of Duchenne muscular dystrophy (DMD). The symptoms include in DMD include Muscle weakness, regular falls, walking on tiptoe, difficulty standing up, delayed onset of sitting, etc. Vamorolone is a corticosteroid class drug, and the formulation present in the market is named Agamree. And the administration form of this formulation is oral suspension.

Copyright & License

Copyright © 2025 Authors retain the copyright of this article. This article is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

BibTeX

@article{180932,
        author = {bhosale ayush and Kawade Madhuri and Rathod Krushna and Hatwar Nandini},
        title = {TO TREAT DUCHENNE MUSCULAR DYSTROPHY WITH AGAMREE (VAMOROLONE)},
        journal = {International Journal of Innovative Research in Technology},
        year = {2025},
        volume = {12},
        number = {1},
        pages = {3442-3458},
        issn = {2349-6002},
        url = {https://ijirt.org/article?manuscript=180932},
        abstract = {The dystrophin protein, which is necessary 
for safeguarding muscle cells during contraction, is not 
produced in Duchenne due to mutations in the DMD 
gene. 
Patients 
experience increasing muscular 
degeneration and atrophy as a result. Over time, chronic 
inflammation plays a role in the development of this 
damage. For individuals with Duchenne muscular 
dystrophy (DMD) who are two years of age or older, the 
dissociative steroid agamree (vamorolone) is used to 
maintain muscle function and reduce inflammation. 
Santhera Pharmaceuticals sold the rights to market 
Agamree in North America to Catalyst 
Pharmaceuticals. The treatment was first created by 
ReveraGen BioPharma. Current clinical management 
guidelines state that mechanical ventilatory assistance 
is typically started at night to address hypoventilation 
and sleep-related breathing issues sometime in the 
second to third decade of a patient's life. All patients 
eventually require help breathing, even throughout the 
day, to survive as their respiratory muscles continue to 
degenerate. Because of the disease's morbidity and 
mortality, DMD is linked to a significant burden on 
afflicted individuals, unpaid caregivers, and society at 
large. Mainly, the DMD is caused by a genetic mutation 
in the DMD gene causes dystrophin, a protein necessary 
for muscle function, to be absent, leading to progressive 
muscle weakness and degeneration. This is the cause of 
Duchenne muscular dystrophy (DMD). The symptoms 
include in DMD include Muscle weakness, regular falls, 
walking on tiptoe, difficulty standing up, delayed onset 
of sitting, etc. Vamorolone is a corticosteroid class drug, 
and the formulation present in the market is named 
Agamree. And the administration form of this 
formulation is oral suspension.},
        keywords = {DMD, Agamree, corticosteroid,  Vamorolone, address hypoventilation.},
        month = {June},
        }

Cite This Article

  • ISSN: 2349-6002
  • Volume: 12
  • Issue: 1
  • PageNo: 3442-3458

TO TREAT DUCHENNE MUSCULAR DYSTROPHY WITH AGAMREE (VAMOROLONE)

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