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 BBC, Mats Steen passed away at 25 years old due to complications from his Duchenne muscular dystrophy. Upon his death,
According to a study published in the Orphanet Journal of Rare Diseases, a study that was intended to identify a new approach to monitoring the progression
Duchenne Muscular Dystrophy Duchenne muscular dystrophy (DMD) is a rare, progressive, and fatal condition. It primarily affects males (1 out of every 3,600 to 6,000
Press Release: uniQure Announces First Patient Treated in HOPE-B Pivotal Trial of AMT-061 in Patients with Hemophilia B
Full Patient Enrollment in Study Expected by Year-End 2019 LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Feb. 04, 2019 — uniQure N.V. (NASDAQ: QURE), a leading gene therapy
What is Duchenne Muscular Dystrophy? Duchenne muscular dystrophy (DMD) is a rare genetic condition that is ultimately fatal. It causes muscles to weaken and usually results
According to a story from pm360online.com, the life sciences company Solid Biosciences has released some preliminary findings from its Phase 1/2 clinical trial of SGT-001.