The European Society for Dermatological Research (ESDR) meeting was held in Lisbon at the beginning of September. On the first day, a session was dedicated to the patients and researchers and the synergies created to go forward on this collaborative path.
After great insight from the organisations working for Pachyonychia, Ichthyosis and Albinism, there was time for Epidermolysis bullosa. Dr Fernando Larcher, senior researcher in EB in Spain, gave an exhaustive talk about the latest advances in research and new therapies for people with Butterfly Skin. He gave details on the in vivo therapy developed by Krystal Biotech and approved by the FDA this year in the USA, the genetic therapy VYJUVEK. He also explained the ex vivo therapies currently in Phase 3 and his lab research focused on exon 80 mutations in the COL7A1 gene. In this regard, he told the audience that after receiving the orphan drug designation a few years ago, they have been working to develop a therapy based on skin equivalents made of corrected skin cells. They are working towards starting a clinical trial in a few patients in 2025-2026.
Afterwards, Dr Núria Tarrats, an expert on EB research at DEBRA Spain talked about the involvement of people with EB and their families in research. After going through the main benefits of including patients in research for the researchers and for the patients themselves, she talked about the importance of having a close relationship with the researchers and the need to establish good communication to manage expectations. Moreover, she talked about other types of research where people with EB and their families can actively participate giving their time and knowledge about EB and its burden, or ask questions about the disease.
Finally, Tarrats explained the initiative DEBRA Spain took, more than 10 years ago, developing a PPS, or priority study for EB research. In 2013 patients, clinicians and researchers listed the priorities for EB research. The top issues focus on symptom relief rather than new treatments, highlighting the need to listen to patients' and families' opinions in this matter. This year, DEBRA UK has launched a new priority study to help mold future EB research priorities and ensure that the voice of the EB community is heard and considered.
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