Tuesday, 23 July 2019 02:44 GMT
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Gene-based medicine for treatment of inherited blindness in UAE




(MENAFN - Khaleej Times) Dr. Mohamed Salim Al Olama, Under-Secretary of the Ministry of Health and Prevention, MoHAP, has revealed the registration of "Luxturna", an innovative gene-based medicine for the treatment of blindness caused by inherited retinal disease.

The medicine's registration comes as part of MoHAP's innovative mechanism for its accelerators to evaluate and approve the world's breakthrough drugs in accordance with the 'Fast Track/Accelerate Process' system.

Al Olama, who is also the Vice Chairman of the Higher Committee for Drug Pricing and Registration, said that the the registration of Luxturna comes in line with the Ministerial Decree No. 28 of 2018 which outlines the mechanism for registering breakthrough and rare medicines.

"It's also part of the UAE's ongoing support for the global initiative, 'Vision 2020: The Right to Sight', launched by the World Health Organisation, WHO, to eliminate avoidable blindness by the year 2020," he added.

"We are witnessing a significant turning point with the expectation of experts that gene-based therapies will be expanded to include other incurable diseases. For that reason, the UAE has approved this new treatment as it paves the way towards future prospects for gene-based therapies which would help patients recover from a lot of complex diseases," Al Olama noted.

The approval of the UAE for this treatment is the third globally at a health authorities level. This comes in line with MoHAP's strategy to build quality and safety for therapeutic, healthcare and pharmaceutical systems according to international standards, as well as the UAE Vision 2021.

For his part, Dr. Mohamed Ezz El Din, Country Manager, Novartis, said, "We received some good news that MoHAP completed the registration process of Luxturna which is the first gene-based therapy in the UAE. We are looking forward to further strengthening our presence in the region to ensure that patients could get this global therapy that may change their lives.

"For those born with a mutation in both copies of the RPE65 gene, nearly 60 percent of patients have severe forms of the disease, with severe visual impairment occurring shortly after birth. RPE65 inherited retinal disease is a rare one, as mutations in both copies of the RPE65 gene affect approximately one in 200,000 people, however, mutations in both copies of the RPE65 gene can lead to blindness. Hence the importance of this innovative medicine, Luxturna, that restores sight and improves vision," added Dr. Ezz El Din.


What is gene therapy?

Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient's cells instead of using drugs or surgery.

Researchers are testing several approaches to gene therapy, including:

- Replacing a mutated gene that causes disease with a healthy copy of the gene.
- Inactivating, or "knocking out," a mutated gene that is functioning improperly.
- Introducing a new gene into the body to help fight a disease.??Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective.

Gene therapy is currently being tested only for diseases that have no other cures.

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Gene-based medicine for treatment of inherited blindness in UAE

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