Today, we received fantastic news! EspeRare and Pierre Fabre announced that the ER-004 clinical trial is posted on ClinicalTrials.gov. The study name is EDELIFE. That means that soon they will launch a pivotal, clinical trial for x-linked hypohidrotic ectodermal dysplasia (XLHED).
The goal of the trial is to reproduce and confirm the results that Prof. Dr. Holm Schneider had when he treated five males affected by XLHED in utero with compassionate use of ER-004. Three babies have normal sweat function as well as other positive results. We are awaiting results on the other two babies.
If successful, this therapy will be the first drug to improve the main symptoms of XLHED! It will be a single course of treatment, with three injections given before birth.
In the coming weeks, we will share with you more detailed information about the clinical trial, including study sites and how to determine if your unborn son affected by XLHED is a candidate.
In 1989, our NFED community first began supporting the XLHED research that led to this moment and have led the effort ever since. We have been patiently waiting for this wonderful news since April of 2018 when we learned that ER-004 had been successful. Research always takes time, which can be frustrating when we are anxious for progress!Read XLHED Research History
But, as promised, we have been committed as a Foundation to doing anything we can to advance this research. We are proud to be working closely with EspeRare and Pierre Fabre and will keep you apprised of clinical trial progress.
Join us in celebrating this major milestone for XLHED research and our NFED family! Stay tuned for more information to come soon.Read Details on Trial
Ectodermal dysplasia can cause a lifetime of challenges. By supporting research, you expand early diagnostics, treatments, pathways toward cures… and hope!
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Ectodermal dysplasia can cause a lifetime of challenges. By supporting research, you expand early diagnostics, treatments, pathways toward cures… and hope!Donate to Boost the Cure