Second Annual International Congress on Advanced Treatments in Rare Diseases
The first International Congress on Advanced Treatments in Rare Diseases, otherwise known as RARE2018, was held in London, England. Now, the second annual congress, RARE2019 will take place next week, March 4th and 5th in Vienna, Austria.
100 people will be in attendance including rare disease patients, researchers, scientists, and pharmaceutical executives. There will be 27 speakers, representing all of these groups of attendees. The conference will cover a wide range of diseases including Gaucher disease, Spinal muscular atrophy (SMA), and Parkinson’s disease to name a few. Three awards will be presented to worthy abstracts.
Some topics that will be covered in this conference include:
- Digital health
- Funding for rare disease research
- Ethical dilemmas in rare disease (Distributive justice/value of life)
- Biobanking/Big data
- Designing clinical trials (Double-blind, placebo-controlled is not always right)
- Challenges in rare disease drug development
- Transitioning from pediatric to adult care in rare diseases
- Mental health within rare disease (Due to wrong diagnoses, delay in diagnoses, prognosis, or an uncertain future)
- Gene therapy
The Overarching Issue
The main purpose of this conference is to emphasize for the entire medical community, the importance of considering rare diseases. This means considering them during diagnosis, in research, and in patient care.
Medical professionals are trained to NOT think of rare diseases. Even if they’ve heard of and are knowledgeable of a rare condition, they may immediately dismiss it because it is unlikely. If someone is sick, there is a much greater chance that they have a common ailment than something rare. Unfortunately, this reality can have a tragic impact on those who are living with a rare condition.
Ari Zimran, who will be a speaker at RARE2019, has strong feelings on this issue. He is the director of the world’s largest Gaucher Clinic, located in the Shaare Zedek Medical Center in Jerusalem. He explains that even though in Israel, Gaucher disease is much more common than in other parts of the world (one in every 850 Ashkenazi Jews have the condition) there is still a severe lack of awareness of the ailment in the country.
There are 7,000 known rare diseases. Of these, only 5% have effective treatments.
Additionally, most patients deal with a significant delay in diagnosis. This delay can cause irreversible complications, significantly hindering the quality of life of patients.
Since most rare diseases are genetic, a delay in diagnosis for one child can also impact the diagnosis of their siblings. Sooner diagnosis could mean better outcomes for not only the patient, but for other members of their family.
Looking Ahead
Every conference, like RARE2019, helps to spread awareness of rare diseases.
Other upcoming conferences you may be interested in may include:
- American Conference on Advanced Treatments in Rare Disease – June 13th-14th, East Brunswick, New Jersey
- Recent Advances in Rare Diseases– June 20th-22nd, Bogotá, Colombia
You can read more about this upcoming conference here.
