2. Good Afternoon My name is Iris Bassi and I have great pleasure in welcoming you all to this inaugural European LAM Scientific and Patient Conference I would especially like to thank all the speakers, researchers and clinicians who have travelled from around the world and from within Italy, to Udine to present their work at the meeting this weekend.

3. On behalf of the organising committee of LAM Italia, I would particularly like to thank the sponsors who have so generously supported us and made this very important project possible It is also with much gratitude, that we thank all LAM patients and the leaders of the international LAM patient organisations who are here from Europe, the United Kingdom, United States of America and Australia and New Zealand

4. It is our challenge and our mission to work to find a cure for LAM in the fastest way possible

5. Lymphangioleiomyomatosis (LAM) is a rare progressive and seriously disabling and potentially fatal illness affecting women Symptoms are shortness of breath, pneumothoraces, haemoptasis, and in some cases, renal angiomyolipomas Progressive decline in respiratory capacity necessitates oxygen therapy in advanced stages and lung transplantation may be an option at end stage disease

6. LAM has been classified as an ‘orphan disease’, being a disorder where the incidence is not more than 1 case per 2000 of the population as defined by the European Union’s definition of rare disorders The World Health Organisation estimates that there are between 7,000 and 8,000 diseases affecting between 6 and 8% of the world’s population Despite rare diseases being characterised by low prevalence, the total number of those affected by a rare disorder in the European Union is between 27 and 36 million people …including approximately 2 million people in Italy alone

7. We do not know precisely how many patients are affected by LAM in the world today as the disease is often misdiagnosed as asthma or emphysema or other unusual breathing disorders Diagnosis can still be a long and frustrating process and in some cases patients are unable to get a diagnosis at all

8. The rarity of LAM and the difficulty of being able to rely on an appropriate diagnosis leads invariably to other difficulties :  Scarcity of information in health institutions  Lack of availability of effective treatment or participation in clinical trials  Reduced availability of clinical data  Difficulty in funding research that could lead to a cure  Reduced availability of tissues and organs for researchers  Psychological and financial burden on LAM patients and their families

9. What can we do?

10. We can collaborate together to make LAM more visible by disseminating results of scientific discoveries and by facilitating the study of methods for early diagnosis and potential effective treatments

11. We can participate in international clinical drug trials

12. We can facilitate the collection of tissues and have them transported rapidly to research centers

13. We can continue to promote national and international LAM research meetings

14. We can open more doors to ensure an enhanced multi disciplinary approach to accelerate involvement by experts from various disciplines

15. We must support, strengthen and accelerate cooperation among basic science researchers, clinicians and patients in order to avoid repetition and wastage in this vital process of discovering the cure for LAM

16. There is a saying that “many times the rocky road is the road of growth, transformation and empowerment” If we can find the willingness to look from a positive vantage point, almost any challenge can offer an opportunity to grow and understand

17. LAM patients have a rocky and challenging road to face, but our future is in our hands; with our participation, we can support the scientific community When we have the courage to accept the obstacles and look at this disease squarely in the face, or when we ask for help when we are afraid, or when we participate at meetings and speak about LAM…..this is the way the rocky road helps us to evolve…until the day we arrive at the solution

18. There are many reasons to be optimistic !!!

19. We have among us the best researchers in the world; scientists who are dedicating their lives to discover a treatment and ultimately the cure for LAM We have many highly efficient LAM patient organisations working hard every day to raise funds and support research Above all, we are living in the era of the “DNA Revolution” The Human Genome; the complete copy of the entire set of human gene instructions was completed in 2003….. human genetic mapping information is there for researchers to use Above all, we are living in the era of the “DNA Revolution” The Human Genome; the complete copy of the entire set of human gene instructions was completed in 2003….. human genetic mapping information is there for researchers to use

20. Researchers have discovered what some of our genes do and have found that some mutations like the TSC 1 and TSC 2 genes, are associated with disorders such as LAM. Continuing research is vital and we must support it on all levels LAM research can also teach us much about diagnosis and treatment of other diseases too; thanks to research, terrible diseases including some cancers are nowadays treatable

21. Not all diseases are yet curable, but we can say that thanks to the enormous progress of science, some of these may become treatable in the future

22. In this exciting environment, we invite all participants to be creative and critical, to ask questions to the speakers and to participate actively in all sessions

23. Thank you all for being here for taking the opportunity for discussion, and enrichment over the next two days and most of all for helping to take us a further step forward in combating the complex maze of LAM

24. Thanks! from Associazione LAM Italia Onlus