Gene Therapies - Success in treatment of SMA brings hope for children with rare diseases

News Stories

The Research Australia (INSPIRE) winter issue features Gene Therapy success for infants with Spinal Muscular Atrophy (SMA).

After 25 years of incremental development the possibility of treating genetic diseases by repair or replacement of faulty genes is finally becoming a reality. A stunning example of this approach, known as gene therapy is the successful treatment of infants with SMA.

The SMA pilot program was supported and funded by Luminesce Alliance.

 

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Luminesce Alliance acknowledges the Traditional Custodians of country throughout Australia and their connections to land, sea and community.

We pay our respect to their elders past and present and extend that respect to all Aboriginal and Torres Strait Islander peoples today.

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Luminesce Alliance acknowledges the Traditional Custodians of country throughout Australia and their connections to land, sea and community.

We pay our respect to their elders past and present and extend that respect to all Aboriginal and Torres Strait Islander peoples today.

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