Abstract

CEVALLOS SOLORZANO, Gabriela et al. My daughter, my life / Tu Vivras ma fille (2018) by Gabriel Aghion, from family medicine to biotechnology. Rev Med Cine [online]. 2022, vol.18, n.1, pp.15-27.  Epub June 20, 2022. ISSN 1885-5210.  https://dx.doi.org/10.14201/rmc.27911.

We can see depicted the reality lived by families of children with rare diseases in the movie "My daughter, my life" (Tu vivras ma fille, 2018) by Gabriel Aghion. The first daughter of a marriage is born with Sanfilippo syndrome, an unusual genetic pathology which affects functionality in children from 3 years up and for which there is no effective treatment. This movie, based on a real life story, that show us the effort of some people in search of new treatment options to improve the life of the loved ones, and in that quest, they have developed a treatment based upon the last biotechnological findings, gene therapy, that decreases symptoms from the pathology itself. In this article we are focusing on the search of a treatment based on the existing biotechnological tools, in addition of the social and affective impact suffered by the people and families living these types of illnesses.

Keywords : rare diseases; mucopolysaccharides; biotechnology; gene therapy; Sanfilippo syndrome.

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