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
For years FibroGen has been investigating a drug called pamrevlumab. They believe it could be an effective treatment for Duchenne muscular dystrophy (DMD), pancreatic cancer,
According to a story from CRWE World, the genetic medicines company Audentes Therapeutics has recently announced its intent to develop new therapies for myotonic dystrophy
According to an article from ReliaWire, a sizable portion of the global population is likely to have an immunity to the inert viruses scientists use
Company Announces Plans to File for Conditional Marketing for Duchenne Muscular Dystrophy Drug in the EU
According to a story from Market Screener, the specialty pharmaceutical company Santhera Pharmaceuticals recently released an announcement in which the company revealed its intent to
According to a release from Sangamo Therapeutics and published by PR Newswire, experimental severe hemophilia A drug SB-525 proved to be well-tolerated and generally safe
Females vs. Males with Fabry Fabry disease is an X-linked condition caused by a malfunctioning GLA gene. It was long believed to only affect men.