Today, we received fantastic news! EspeRare and Pierre Fabre announced that the ER-004 clinical trial is posted on  The study name is EDELIFE. That means that soon they will launch a pivotal, clinical trial for x-linked hypohidrotic ectodermal dysplasia (XLHED).

A doctor is shown with three boys affected by x-linked hypohidrotic ectodermal dysplasia. The boys are playing with toys.
Dr. Holm Schneider treated twins, Maarten and Linus (left) in utero with ER-004. The boys developed working sweat glands and had other improved symptoms. Their brother is shown at right.

The goal of the trial is to reproduce and confirm the results that 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.

Our Commitment

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

Shape Our Futures With Research

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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4 comments on “XLHED Clinical Trial One Step Closer to Launching”

  1. 1
    Dottie Soliz on July 29, 2021

    This is fantastic news, I’m 67 female with the disorder since birth no hair or nails I will volunteer for testing or on line questioners with regards to living with this life long problem I have one child effected other 2 we’re ok

    1. 2
      Kelley Atchison on July 29, 2021

      Hi, Dottie. We share in your joy! Thank you for your interest in participating in ectodermal dysplasia research and data collection. Any future research projects on XLHED or other types of ectodermal dysplasias will be posted to our website and social media. Please contact our office with any questions at 618-566-2020 or

      Be merry and well,
      Kelley Atchison
      Director, Family and Community Programs

  2. 3
    prudhvi kakuru on August 3, 2021

    This news shedded a light of hope for future generations.
    I was waiting for this kind of news every morning.
    Thank you for your research.

    1. 4
      Kelley Atchison on August 3, 2021

      We share in your excitement!

      Kelley Atchison
      Director, Family and Community Programs

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