The conference creates a space for dialogue between science, the clinic, the healthcare system and patient organizations, focusing on the key question: “why so many rare disease therapies do not reach patients, and how to change it.”
When and where
13 March 2026; MUW Didactic Center (2a Ks. Trojdena Street, Warsaw), Hall B, 8:30 a.m. to 5:00 p.m.
Why take part
Conference participants will gain:
- the opportunity to listen directly to the Nobel Prize winner's lecture and participate in an expert conversation,
- current knowledge about barriers and opportunities in the development of therapy for rare diseases,
- the perspective of patient organizations as partners in scientific research,
- an opportunity to exchange experiences between scientists, physicians and patients,
- access to panel and poster sessions presenting Polish and international research initiatives,
- the possibility of networking in the academic, clinical and patient environment.
About Victor Ambros, the Nobel Prize winner
In 2024, Professor Victor Ambros together with Professor Gary Ruvkun were awarded the Nobel Prize in Physiology or Medicine for the discovery of microRNAs and their role in post-transcriptional gene regulation. The discovery of a new class of RNA molecules in 1993 fundamentally changed the understanding of gene expression control mechanisms. Prof. Ambros showed that microRNAs regulate gene expression by deciding which mRNAs are converted into proteins. MicroRNA acts as a key, previously unknown, mechanism of "fine-tuning" in cells. It is now known that microRNA molecules play an important role in the development of numerous human diseases, including tumors, neurological disorders, and metabolic diseases. The discovery was made during research on the nematode C. elegans.
Professor Victor Ambros was born in 1953 in Hanover, New Hampshire, and is a researcher at UMass Chan Medical School in Worcester, Massachusetts. Professor Craig C. Mello, a Nobel laureate who visited the MUW last year, works at the same university. Professor Victor Ambros's father, Longin, was born in the Vilnius Region. He emigrated to the United States after World War II.
Prof. Ambros has been the chairman of the Scientific Council of the International Institute of Molecular Mechanisms and Machines (IMol) of the Polish Academy of Sciences since 2025. In March, he will come to Poland for another meeting of this advisory body. In addition, Prof. Ambros is an ambassador of the patient research foundation AGO Alliance Poland, which develops treatment for AGO syndrome. In addition to the Nobel Prize, he received the 2008 Lasker Award, the Wolf Prize in Medicine (2014) and the Breakthrough Prize in Life Sciences (2015).
Registration for the conference
- Mandatory registration form website
- Participation in the conference sessions is free of charge.
- Seats are limited – pre-registration is required.
IMPORTANT INFORMATION FOR MUW STUDENTS AND DOCTORAL STUDENTS: In accordance with the ordinance No. 53/2026 of MUW Rector, from 9 a.m. to 12 noon on March 13, 2026, rector's hours will apply to students and doctoral students of the Medical University of Warsaw – participants of the Polish Day of Rare Diseases conference with the Nobel Prize winner, Prof. Victor Ambros.
QR code to register for the event:
Poster session and active participation
The following are invited to actively participate in the conference:
- scientists and research teams working in the field of rare diseases,
- patient organizations that want to present their activities, research projects or patient-supporting initiatives.
- it is possible to:
- submit a scientific poster or a patient organization poster,
- send a short video (1–2 minutes) by patient organizations presenting their mission and activities – selected materials will be presented during the conference.
Detailed information on applications is available in the registration form.
Organizers
Medical University of Warsaw, IMOL – International Institute of Molecular Mechanisms and Machines, and AGO Alliance Poland.