The ED Society are proud to continue supporting this research, which is focusing on treatment to correct some symptoms in X-Linked Hypohidrotic Ectodermal Dysplasia.
What’s the latest?
THE FIRST BABY TREATED WITH POTENTIAL RECOMBINANT PROTEIN THERAPY IN THE U.S. HAS BEEN BORN
ST. LOUIS, May 31, 2023 – A baby boy born this month is the first to receive a recombinant protein therapy in the United States that may correct the symptoms of his rare disorder.
You can read the full press release here
We often get asked “Why is it always the boys?” Boys affected by XLHED exhibit the full symptoms
So, why is the EDELIFE clinical trial just for XLHED boys?
This is the news we have all been waiting for…..
Clinical sites are now open in France, Germany, Spain and Italy to recruit participants for the EDELIFE clinical trial.
Women who know or suspect that they are XLHED carriers and who are pregnant with a boy are invited to consider participating in the study. They can reach out to the clinical sites and they do not have to live in those countries to participate.
“Rare diseases, such as XLHED, often don’t receive the attention and research investments that patients and their families deserve. The FDA’s decision to grant Breakthrough Therapy Designation to ER-004 for the prenatal treatment of XLHED represents a significant milestone for patients, and EspeRare alike, in highlighting the severity of this condition and the importance of offering a potential first therapeutic option to this underserved patient population,” said Caroline Kant, EspeRare’s Executive Director.
“This innovative treatment approach has the potential to fundamentally change the lives of these patients, and may also pave the way for other prenatal treatments to correct genetic conditions before birth. We look forward to continuing to work closely with the XLHED patient community towards these goals.”