An individual suffering Gene Therapy for Rare Disease can receive a targeted, specialized treatment in which corrected genes are inserted.

This may entail administering the medication or medications frequently when managing a chronic disease. Gene therapy, on the other hand, has the ability to address fundamental genetic flaws, providing a cure as opposed to only treating symptoms.

Since more than 80% of uncommon diseases have a single-gene known aetiology, gene therapy is very important for people with rare disorders. Instead of treating the disease, traditional small molecule medications frequently function by reducing symptoms. Gene Therapy for Rare Disease may entail administering the medication or medications frequently when managing a chronic disease. Gene therapy, on the other hand, has the ability to address fundamental genetic flaws, providing a cure as opposed to only treating symptoms.

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