New Muscular Dystrophy Treatments 2021



Article last updated on 4/1/2021

What is Muscular Dystrophy (MD)?

Muscular Dystrophy (MD) refers to a group of over 30 inherited genetic and progressive diseases caused by a mutation in a person’s genes. The different types of MD vary in signs, symptoms and affect specific muscle groups. Some types of MD are visible in infancy while others may appear in middle age or adulthood.1

Duchenne Muscular Dystrophy (DMD) is the most prevalent neuromuscular disorder, affecting up to 1/3600 male births worldwide. There is currently no known cure for Duchenne Muscular Dystrophy (DMD). Treatment aims to control symptoms to improve quality of life. 2


How is Muscular Dystrophy (MD) treated?

There are several approved Muscular Dystrophy (MD) medications available depending on the form of the diseases. Here are some of them:


Viltepso (viltolarsen)3

Viltepso (viltolarsen) is an antisense therapy indicated for the treatment of Duchenne Muscular Dystrophy (DMD) with a confirmed deficiency of the dystrophin gene amenable to exon 53 skipping therapy.

Viltepso (viltolarsen) was approved for the treatment of patients with Duchenne Muscular Dystrophy (DMD) who are amenable to exon 53 skipping therapy by:

  • The Food and Drug Administration (FDA), USA on August 12, 2020.
  • The Pharmaceuticals and Medical Devices Agency (PMDA) under the name Viltolarsen (NS-065/NCNP-01), Japan, March 25, 2020.

The FDA has granted accelerated approval to Viltepso, and granted it with Priority Review, Fast Track, Orphan Drug and Rare Pediatric Disease designations.

The PMDA has granted Viltolarsen with "Sakigake” designation (i.e. the Japanese version of Breakthrough Therapy Designation), Orphan Disease designation, and designation of Conditional Early Approval System.


Vyondys 53 (golodirsen)4

Vyondys 53 (golodirsen) is an antisense nucleotide 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 53 skipping.

Vyondys 53 (golodirsen) was approved for the treatment of DMD patients with a confirmed mutation of the DMD gene that is amenable to exon 53 skipping by the Food and Drug Administration (FDA), USA, on December 12, 2019.

The FDA approved Vyondys 53 (golodirsen) under accelerated approval.


If you are trying to access a Muscle Dystrophy treatment that is approved outside of your country of residence, we might be able to help you access it with the help of your treating doctor. You can read more about the medicines we can help you access and their price below:




Why access treatments for Muscular Dystrophy (MD) with TheSocialMedwork?

TheSocialMedwork is registered in The Hague with the Dutch Ministry of Health (registration number 6730 BEM) as an independent medicines intermediary. We have helped patients from over 85 countries to access thousands of medicines including. With a prescription from your treating doctor, you can count on our expert team to safely and legally guide you to access medications for Muscular Dystrophy (MD). If you or someone you know are looking to access a medicine that is not yet approved where they live, we can support you. Contact us for more information.


References:

  1. Cdc.gov
  2. Nhs.uk
  3. Viltepso (viltolarsen) - Thesocialmedwork.com<
  4. Vyondys 53 (golodirsen) Thesocialmedwork.com

Disclaimer: This article is not meant to influence or impact the care provided by your treating physician. Please do not make changes to your treatment without first consulting your healthcare provider. This article is not intended to diagnose or treat illness or to influence treatment options. TheSocialMedwork is as diligent as possible in compiling and updating the information on this page. However, TheSocialMedwork does not guarantee the correctness and completeness of the information provided on this page.