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Priapism commonly occurs in men with sickle cell disease, and the risk increases with age. ... As you can see, some of these are within your control, while others are related to genetics or illness.
In 2023, the first drug making use of CRISPR gene editing, Casgevy, was approved for use in the United Kingdom to cure sickle-cell disease and beta thalassemia. [13] [14] Casgevy was approved for use in the United States on December 8, 2023, by the Food and Drug Administration. [15]
It treats sickle cell disease (SCD) and beta thalassemia, both rare inherited illnesses that affect the function of a person's red blood cells. ... One of those programs is a clinical-stage gene ...
Fragile X syndrome (FXS) is a genetic neurodevelopmental disorder characterized by mild-to-moderate intellectual disability. [1] The average IQ in males with FXS is under 55, while about two thirds of affected females are intellectually disabled. [3][4] Physical features may include a long and narrow face, large ears, flexible fingers, and ...
For example, sickle-cell anemia is a human genetic disease that results from a single base difference within the coding region for the β-globin section of hemoglobin, causing a single amino acid change that changes hemoglobin's physical properties. [78]
Inaxaplin is already in a phase 3 trial for treating APOL1-mediated kidney disease. This indication would open up an even bigger market than CF to Vertex if the experimental drug wins approval.
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