Remember the Girls
Females who are affected by x-linked recessive genetic disorders have traditionally been deemed to be “just carriers” who are completely unaffected by the disorder. However, for many x-linked recessive disorders (which in actuality are not truly recessive), this is simply untrue. In addition to the fact that female carriers may pass the disorder along to their offspring, many female carriers may develop physical symptoms of the disorder, however the medical community often fails to connect these symptoms to their carrier status, subjecting these women to many years of physical problems they should not have to tolerate. This can be frightening and lonely.
There has long been a gender bias in medicine, with males being predominately enrolled in clinical trials. Most studies that did include women did not report results by gender. Although this has begun to change in recent years, there is still an enormous lack of scientific research and understanding when it comes to carriers of x-linked disorders. Remember the Girls works to create a community of females affected by x-linked disorders to break away from the “carrier” stigma. While it is extremely important to continue searching for treatments and cures for males with x-linked disorders, it is also important to study the effect of these disorders on female carriers, so they too have the opportunity to live a full and healthy life.
Condition Awareness & Advocacy
Here is a list of conditions this partner raises awareness and advocacy for:
Remember the Girls
Patient Worthy Posts on X-Linked Genetic Disorders
According to a story from MD Edge, a phase 2 clinical trial testing the drug concizumab as a treatment for hemophilia A and hemophilia B
According to a story from globenewswire.com, the drug company Amicus Therapeutics recently announced that Argentina’s Agency of Medicines, Food, and Medical Devices (ANMAT) has granted
Advocates for Duchenne Muscular Dystrophy Start Company Designed to Collect Data Directly From Patients
According to a story from Xconomy, the US Food and Drug Administration (FDA) made a controversial decision three years ago when it approved the drug
According to a publication from Fabry Disease News, biotechnology company Avrobio recently announced that early phase 1 and 2 clinical studies of experimental Fabry treatment
According to a story from health.economictimes.indiatimes.com, the country of India is currently beginning to take more steps in order to address the dire unmet medical