Advancing treatments for rare diseases together

September 2024

24Sep

Second site open in the USA for EDELIFE Clinical Trial

XLHED, rare disease, EDELIFE clinical trial

We are thrilled to announce the opening of a new clinical site in the United States, bringing the total number of sites for the EDELIFE clinical trial to eight, spanning six countries. Cedars-Sinai Medical Center in Los Angeles, California, has been chosen as a second location to complement the existing site at Washinghton University in St Louis, Missouri. By adding a new location, we aim to make the Edelife study more geographically accessible and provide more opportunities for potential patients to participate.  

In partnership with Pierre Fabre, EspeRare Foundation is leading this groundbreaking study, which has been welcomed by healthcare professionals and patient communities. The EDELIFE clinical trial aims to address a significant unmet need, as there is currently no curative treatment available for XLHED (X-linked hypohidrotic ectodermal dysplasia).

Recruitment is ongoing across Europe and the USA to enroll women who know or suspect they are carriers of XLHED and are pregnant with a boy. Clinical sites are now open in the UK, Germany, France, Italy, Spain, and the USA.

However, women in any country who meet the criteria may still have the opportunity to join this pivotal study.

https://edelifeclinicaltrial.com/ to find out more

23Sep

Advancing Equitable Genomic Testing in Rare Diseases

In the Q3 2024 issue of Rare Diseases by Mediaplanet, Sharon Terry, CEO of Genetic Alliance and President of EspeRare, highlights the critical need for equitable, patient-centered solutions in rare disease care. “The urgency for equitable access to state-of-the-art genomic-based sequencing has never been more critical,” Sharon states. She passionately advocates for inclusive research and collaboration to address the unmet needs of underserved populations. Sharon’s vision extends through iHope Genetic Health, a groundbreaking Genetic Alliance program offering free genome and exome sequencing for children suspected of genetic diseases, providing accurate diagnoses for better follow-up care.

You can read the full article on page 8 posted here.

To find out more about iHope Genetic Health click here.