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Lawmaker giving hope to Floridians with kids with rare diseases after losing his 4-year-old sonPINELLAS COUNTY, Fla. (WFLA) — There are more than 7,000 known rare diseases that affect 350 million people across the globe. One local State Representative sponsored a law that is giving a ...
In one particular variant of this disease known as mucopolysaccharidosis I (MPS I), a deficiency of the enzyme alpha-L-iduronidase causes a build up of GAGs in tissues and organs, which in turn ...
Carrying Tay-Sachs disease may protect against tuberculosis (TB). In Ashkenazim populations, up to 11 percent of the people are Tay-Sachs carriers. During World War II, TB ran rampant in Eastern ...
Tay-Sachs disease, the more common of the two disorders, typically manifests within the first year of life, progressing quickly and often proving fatal within a few years. In rare cases ...
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