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
A Greater Number of Drugs Are Getting Cleared With the FDA’s Accelerated Approval. What Does This Mean?
According to a story from Boston Health News, The US Food and Drug Administration’s Accelerated Approval program is being used to approve a steadily growing
According to a story from The Atlantic, the continual development of gene editing technologies like CRISPR have the potential to cure a substantial portion of
New Updates on Patients Taking Part in Studies of a Gene Therapy for Fabry Disease Have Been Released
Avrobio has released updates on patients in two clinical trials of a potential gene therapy for Fabry disease. According to the announcement, two patients taking
According to a story from statnews.com, the drug developer Sarepta has recently announced the its clinical trial testing an experimental gene therapy has resumed after
The first US patient has been dosed with the drug MIN-102 as part of a clinical trial into its effects in patients with adrenomyeloneuropathy. For
The non-profit organisation CureDuchenne has announced that they have made a $500,000 investment in the company 4D Molecular Therapeutics in order to support the development