We recognize that coping with a rare genetic condition like Alport syndrome can present many challenges. Because Alport syndrome affects the kidneys, people living with the condition experience gradual loss of kidney function. This may lead to end-stage kidney disease that requires dialysis or kidney transplant. Other symptoms, such as loss of hearing and vision, can occur during late childhood or early adolescence.
Because Alport syndrome symptoms can worsen over time, it is important to not ignore them. Staying informed and seeking the right support can help ensure your well-being.
If you qualify to participate you will receive the investigational drug as an injection under the skin on a weekly basis and for approximately 2 years. The first year 30 patients receive active investigational drug and 15 patients receive placebo (inactive medicine). The second year every patient receives active investigational drug. Periodic tests and assessments will be performed either at the study center or at home.
More details on study can be found here
Should you qualify and decide to enroll, you will: