Rare diseases are the focus of gene therapy

2023-11-14 16:05:26

The associations “Forum Rare Diseases” and “Pro Rare Austria” organized the Austrian Congress for Rare Diseases for the 13th time

Vienna (OTS) – This year’s congress was entirely dedicated to the latest gene therapies for rare diseases and had the motto “Hope, a look into the future!”.

The location of the congress on October 6th and 7th, 2023 was the Josephinum Medical History Museum in Vienna. This year’s conference program was designed by Univ.-Prof. Dr. Susanne Greber-Platzer and Dr. Vassiliki Konstantopoulou (Medical University of Vienna) and by Pro Rare Austria designed. In addition to almost 20 lectures by international and national experts who presented the latest scientific developments on gene therapies for various rare diseases, lectures by patients and relatives as well as panel discussions were integrated, which attracted the almost 200 participants on site and 100 online with interest and pursued tension.

“In Austria, more than 450,000 people are affected by rare diseases,” said the chairwoman of the Rare Diseases Forum, Univ.-Prof. Dr. Daniela Karall. A “rare disease” is defined as having a frequency of less than 1 in 2,000 affected people; there are currently between 6,000 and 8,000 known rare diseases.

Representatives from Austrian university hospitals, Gesundheit Österreich GmbH, the Federal Ministry of Health, the pharmaceutical industry and Pro Rare Austria, among others, discussed the current situation.

“In the panel discussion, the always current topic of financing expensive therapies for rare diseases was discussed constructively with key stakeholders, with the obstacles as well as clear suggestions for solutions being highlighted. “Essentially, it’s regarding expanding funding from a single pot and ensuring a fair, uniform distribution between inpatient and outpatient, also throughout Austria and with the involvement of the respective experts in therapy decisions,” says Univ.-Prof. Dr. Wolfgang Sperl, Rector of the Paracelsus Medical University in Salzburg.

“The focus in 2023 was to shed light on innovations in gene therapy and, in particular, to establish a connection between research and those affected,” says the conference leader, Univ.-Prof. Dr. Susanne Greber-Platzer continues.

“We see the annual congress as an important platform for new ideas, new relationships and, above all, new partnerships,” says Ulrike Holzer, chairwoman of Pro Rare Austria. “It is particularly important for us to actively involve those affected and their families and to hear their experiences first hand. Equally important is our demand that access and reimbursement for the new therapies in Austria must be ensured,” concluded Holzer.

“Hope was the title of the congress, a wish that lies in the future. In my opinion, everyone involved in this congress was able to strengthen hope not only among those present, but among all those affected and allies. The enormous commitment and tireless motivation “, which also became visible in the congress, ultimately aim to improve the future in the field of rare diseases in every respect,” says Vassiliki Konstantopoulou convincingly.

The Austrian Congresses for Rare Diseases are intended to enable networking and encounters between everyone who cares regarding the concerns of people with rare diseases.

“The diagnosis and treatment of rare diseases is one of the success stories of medicine. 20 years ago, topics such as independence, their own family life or job were hardly conceivable for affected children and young people. However, with improved life expectancy, many new challenges arise. These future topics can also be discussed and discussed during the congress,” concludes Dr. Caroline Culen, Managing Director of Austrian League for Child and Adolescent Health.

Questions & Contact:

Rare Diseases Forum
Contact: ao. Univ.-Prof. Dr. Daniela Karall, Chairwoman of the Rare Diseases Forum
[phone]
Daniela. [e-mail]
www.forum-sk.at

Pro Rare Austria, Alliance for Rare Diseases
Contact: Mag. Gabriele Mayr, public relations
[phone]
[e-mail]
www.prorare-austria.org

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