DMD, short for Duchenne muscular dystrophy, is a severe and rare X-linked neuromuscular disorder that occurs in childhood. It is mostly found in males and is caused by the absence of a dystrophin protein. This absence is a result of a mutation of the DMD gene located on the X chromosome’s short arm. The symptoms are usually visible by age 6 and most patients become non-ambulatory by age 13.
The prevalence of DMD in the MENA region is the same as found in the other regions of the world, with 1 case in every 3600 – 6000 live male births. Saudi Arabia leads in efforts made for understanding epidemiological trends of neuromuscular disease, followed by other countries like Egypt, Lebanon, Syria, Jordan, Kuwait, etc.
Facts About The Current DMD Treatments
- Corticosteroids such as deflazacort and prednisone/prednisolone are used to treat more than 50-80% of DMD patients in the MENA region, thus making them the current gold standard treatment.
- In Israel, Sarepta Therapeutics’ Exondys 51 (eteplirsen) and PTC Therapeutics’ Translarna (ataluren) have been approved for the treatment of DMD.
- Translarna and Exondys 51 are the only available drugs in the entire MENA region and were approved in Israel only.
- Drugs like Vyondys 53 and Amondys 45 from Sarepta, are expected to enter the MENA treatment market soon.
Emerging DMD Treatment Landscape
Key companies like Pfizer (PF-06939926), Santhera Pharmaceuticals/ReveraGen BioPharma (vamorolone), Fibrogen (pamrevlumab), Italfarmaco (givinostat), and Sarepta Therapeutics (casimersen, golodirsen) have been conducting studies on developing novel therapies to treat DMD and further delay its progression.
The treatment landscape for DMD is already improving in MENA countries like Saudi Arabia, UAE, Israel, etc. With an increased awareness of DMD in the MENA countries, the diagnosis rate too is expected to improve. Simultaneously, national NBS programs will also help diagnose the disease on time, thus making the treatment even more effective. Additionally, factors like government initiatives for improving market access and encouraging companies to enter their respective countries for clinical trials will further boost the advancements in the DMD treatment landscape of the MENA region.
Conclusion
Awareness about DMD, standardized referral patient registries, and support groups will play a vital role in the better management of the disease. While the MENA region has a few isolated patient support groups, there is limited patient advocacy in the region. For this, ongoing education through different channels is necessary. The social stigma associated with Duchenne Muscular Dystrophy will also be eliminated via such efforts.
Source: Can Headways in Newborn Screening and Emerging Therapies Revamp the Treatment and Diagnosis of Duchenne Muscular Dystrophy (DMD) in the MENA Region? – DUPHAT.
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