Advancing treatments for rare diseases together

February 2023

23Feb

EspeRare is present at the Rare Disease Day at NIH 2023

EspeRare and its partner Pierre Fabre Group will participate in the Rare Disease Day at NIH on 28th February 2023, presenting their ground-breaking clinical study for families with XLHED. The virtual booth will give the possibility to attendees from various parts of the world to connect and find out more about the new treatment paradigm and engage with the speakers.

National Institutes of Health (NIH), through this event aims to raise awareness about rare diseases, the people they affect, and NIH collaborations that address scientific challenges and advance research for new treatments. The event is free and open to the public. Registration is required prior to attending: https://hopin.com/events/nih-rare-disease-day-2023/registration

 

02Feb

Pierre Fabre and EspeRare move forward a pioneering in-utero therapy for XLHED patients

When French pharma company, Laboratoires Pierre Fabre and Swiss not-for-profit organization, EspeRare, joined forces by the end of 2020, their goal was to develop a life-changing treatment for a debilitating rare disease, X-Linked Hypohidrotic Ectodermal Dysplasia, which affects approximately 1 in 25,000 live male births every year.

“Currently, there are no therapeutic options for XLHED. Only the symptoms of the disease can be alleviated” – as Caroline Kant, CEO and co-founder of EspeRare and Caroline Miklaszewski, Project Director at Pierre Fabre, jointly stated.

There is no doubt that the drug development in rare diseases is highly challenging but when two patient-centric organizations combine complementary expertise, the ground-breaking research and a novel therapeutic approach can see a daylight. ER-004, a pioneering in-utero protein replacement therapy, is currently being investigated in the EDELIFE clinical trial and  if approved, it will become the first therapy to address a genetic condition before birth.

To read the full article, please follow the link: Discovery & Development (themedicinemaker.com)