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

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)

PRE – RARE DISEASE DAY – 23.02.2013 – CITY MALL –RAISING PUBLIC AWARENESS

PATIENT STORIES AND INFO ABOUT RARE

DISEASES

Design by Concept marketing agency – Skopje (pro bono)

RARE DISEASE DAY – 28.02.2013 (MEDICAL FACULTY – AMPHITHEATER)

RARE DISEASE DAY – 28.02.2013 (PATIENTS, DOCTORS, MEDIA, GUESTS, SUPPORTERS)

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

LIST OF MEDIA THAT PUBLISHED NEWS ABOUT RARE DISEASE DAY, 28.02.2013Televisions: 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

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

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)

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

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.

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. Aspazija Sofijanova – 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 Alagille Syndrom.

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