About the association

1. Campaign for raising public awareness about rare diseases and strengthening of the association of citizens for rare diseases – Life With Challenges (2013, R. Macedonia) Presentation by Vesna Stojmirova at the Gaucher Advisory Meeting at Waterford, Ireland, 22nd of March, 2013

2. About the association • Founded in 2009 by Gaucher patients and parents • EGA member in 2012 • EURORDIS member in 2013 • PAL award in 2012-2013 • National cooperation with patient associations for hemophilia, cystic fibrosis, CML, HAE … • Balkan cooperation with patient associations in Bulgaria, Slovenia, Serbia, Bosnia and Herzegovina, Croatia. • Honorary member – Mrs. Mary Jo Wohlers - wife of the American Ambassador in Macedonia (former medical nurse)

3. Pre – rare disease day – 23.02.2013 – City Mall –raising public awareness

4. Patient Stories and info about rare diseases

5. Design by Concept marketing agency – Skopje (pro bono)

6. Rare Disease Day – 28.02.2013 (Medical Faculty – Amphitheater)

7. Rare Disease Day – 28.02.2013 (patients, doctors, media, guests, supporters)

8. List of media that published news about Rare Disease Day, 28.02.2013 News agencies: • 1. Mia news agency - http://www.mia.mk/mk/Inside/RenderSingleNews/33/102751474 Newspapers: • 2. Utrinski - http://www.utrinski.com.mk/default.asp?ItemID=2B6F4845A24ACA409A9DAB34C505E4BB • 3. Vecer - http://www.vecer.com.mk/default.asp?ItemID=4BEFACEBB3BCFF4BADE7A154F3B94D37 • 4. Denesen - http://denesen.mk/web/2013/02/28/odbelezuvanje-na-svetskiot-den-na-retki-bolesti/ • 5. Nova Makedonija - http://www.novamakedonija.com.mk/NewsDetal.asp?vest=3113831162&id=9&prilog=0&setIzdanie=22818

9. List of media that published news about Rare Disease Day, 28.02.2013 Televisions: • 6. MTV 1 – Macedonian National Television - http://www.mtv.com.mk/mk/vesti/ticker.css/76321/odbelezhuvanje_na_svetskiot_den_na_retki_bolesti.aspx • 7. And we were guests at a TV show – TUNEL – 50 minutes prime time dedicated to rare diseases • 8. Sitel national television - http://www.sitel.com.mk/kampanja-za-podiganje-na-svesta-za-retkite-bolesti-na-svetskiot-den-na-retki-bolesti • 9. Sitel national television - http://www.sitel.com.mk/odbelezhuvanje-na-svetskiot-den-na-retki-bolesti • 10. 24 news national television and a web portal - http://24vesti.mk/desetici-pacienti-so-retki-bolesti-ostaveni-na-cedilo-od-drzhavata • 11. Also we were guests at a TV show on 24 news – 40 minutes – One on One discussion about rare diseases – available at: http://www.youtube.com/watch?v=vSLTi0c0ogA&list=PL5cM0JDOhCh5nghrluYah4H6_Ui98cZ7m • 12. Alfa national television - http://www.alfa.mk/News.aspx?ID=51683#.UTR9YDCklho • 13. Alsat National television - TV show about health - 30 minutes talking about rare diseases in Macedonia • 14. Kanal 5 national television - http://kanal5.com.mk/default.aspx?mId=-1&egId=13&eventId=107737

10. List of media that published news about Rare Disease Day, 28.02.2013 News web portals: • 15. Kurir - http://kurir.mk/makedonija/vesti/105652-Zivot-so-predizvici--odbelezan-denot-na-retki-bolesti • 16. Kurir - http://kurir.mk/makedonija/vesti/105522-Odbelezuvanje-na-Svetskiot-den-na-retki-bolesti • 17. Tocka - http://tocka.com.mk/1/88540/dvaeset-lica-vo-zemjava-stradaat-od-retki-bolesti • 18. Komunikacija - http://www.komunikacija.mk/se-odbelezhuva-svetskiot-den-na-retki-bolesti/ • 19. Maxim - http://maxim.mk/deneska-kje-se-odbelezhi-svetskiot-den-na-retki-bolesti • 20. Netpress – web portal and news agency -http://www.netpress.com.mk/mk/vest.asp?id=122343&kategorija=1 • 21. Defektologija - http://www.defektologija.org/nastani/odbelezuvanje-na-svetskiot-den-na/ • 22. Nova TV, on line television - http://novatv.mk/index.php?navig=8&cat=18&vest=1124 Weekly magazine • 23. Tea Moderna

12. Further actions for raising public awareness and strengthening of the association • Info brochures in Macedonian language about rare diseases (for patients, families and doctors) • Media event for promoting the brochures at the Children Clinic • Free download of the brochures at the web page of the association (http://challenges.think.mk/) • Further improvement of the web page connected with facebook page and group and you tube channel. • https://www.facebook.com/groups/312483895490987/ • https://www.facebook.com/LifeWithChallengesi • http://www.youtube.com/user/lifewithchallenges?feature=mhee • Development of another brochure with personal stories (doctors and nurses together with patients)

13. Situation of patients with rare diseases in Macedonia – 40 patients • 12 Gaucher patients • 4 on Cerezyme with donation ( 2 children and 2 adults) • 1 on clinical study – Eliglustat (1 adult) • 7 without treatment (4 children, 3 adults) • 6 PKU patients (3 children, 3 adults) • 1 Alagille syndrome (child) • 10 Hereditary angioedema (HAE) (2 children, 8 adults) • 1 Tyrosinemia (child) • 2 with Mucopolisaharydosis (children) - on treatment provided by the Ministry of Health • 8 with other rare diseases

14. Program for Rare Diseases – Ministry of Health – 2009 • European program • Building awareness • Early diagnosis • Orphan drugs • Patient register • Availability to treatment • Estimated 20 patients • Esitmated budget of 452 032 EUR.

15. Progress in negotiation with the Ministry for Health and the Health Insurance Fund in Macedonia • Reforming of the Commission for Rare Disease in Macedonia – chairman – D-r. AspazijaSofijanova – Director of the children clinic in Skopje • Delivering list of treatment needs for patients with rare disease to institutions (mini register of the association) • Negotiation with the Health Fund – acceptance of treatment for patients with PKU and AlagilleSyndrom. • Negotiation with the Ministry – one Gaucher patient (12 year old girl with enormous spleen and liver) • Hope for other patients with rare diseases to start treatment