International Gaucher Day will be marked at the end of October, with an educational lecture for doctors and meeting for families. Details will follow after 20th October.
The citizen association for rare diseases “Life with Challenges” organizes The First Balkan Meeting for Alport Syndrome in Ohrid, Republic of Macedonia from 31st August to 2nd September. The event is organized by Gordana Loleska, Vesna Aleksovska and Velibor Tasich, MD. The event began with an opening addresses from a mother of a child with Alport syndrome, the Minister of Health and the Mayor of Ohrid Municipality.
Statement, Gordana Loleska, activist in LIFE WITH CHALLENGES, mother of a child with Alport syndrome, organizer of the conference:
I am glad that we have here present today institutions, patients and doctors, because together we can achieve more for the families that face a rare disease such as Alport Syndrome. I hope that the families will have the opportunity to talk and to sincerely ask about all they need to know about Alport syndrome in the course of these 3 days. As a mother of a child with Alport syndrome I know what it feels like when the world crashes upon you when you get diagnosis with which you do not know what to do because there is no cure. It remains for us to bear hope that there will be new clinical trials and medicines that will extend and improve life with Alport syndrome.
Statement, Venko Filipche, Minister of Health of R. Macedonia:
I believe that only in such meetings where all the concerned parties are present we can jointly do much more and get good results . Doctors and experts should always be at disposal to patients, as well as to institutions. We will try to improve the life of all patients, citizens of our country. We have started cooperating with the rare disease associations a year ago when we had meetings where all the problems and challenges were genuinely discussed. We have completed some of the tasks, and we are still working on others. The electronic registry has been completed, three new medicines have been procured. Now we are working on improving the diagnostic of rare diseases and procuring new medicines. At a meeting with the Minister of Finance we demanded higher budget for health care as well as for rare diseases. Also, we are working on a special fund with the Chamber of Commerce for creating additional means for medicines for rare diseases.
Statement: Konstantin Georgievski, Vice -Mayor of Ohrid
We are proud to host this Balkan meeting and we are here to support every process of cooperation. I am convinced that during these three days my respected colleagues and experts will offer knowledge and new information that patients and families with Alport Syndrome will find useful and at the same time they will exchange experiences among themselves for further improvement of the treatment of Alport Syndrome in Macedonia. I wish you a pleasant stay in our most beautiful city, Ohrid.
Statement: Vesna Aleksovska, President of the association:
When I first met Gordana Loleska I knew that she was a mother who would fight not only against the disease of her child but also for all the children with rare diseases and she has made it. It is with insurmountable enthusiasm that Ms. Loleska has organized many events around Ohrid to raise public awareness on rare diseases and she has also succeeded to have a post stamp created for rare diseases. I hope that other families will also be encouraged to demand improved quality of life because all citizens deserve a chance for a better life. The Alport Syndrome is a disease for which there is no cure at present; but researchers are carried out and I hope that when it is approved the same will be soon available with us. We must strive towards systematic, long-term solutions that will bring to improve the access to early diagnosis, early treatment and care, and control and follow-up of diseases regardless if they are rare or common.
The present situation with rare diseases in Macedonia
With the creation of the registry and with the increase of finances in the rare disease programme in 2015 and 2016 the procurement of over 29 medicines for 15 different diagnosis was enabled, medicines that until then were not available to patients with rare diseases . 2017 was a year when nothing happened regarding rare diseases. In 2018 we got the electronic registry for rare diseases that should alleviate the registration and follow-up of patients as well as the follow up of the quantity of medicines. This year finally after 7 months we have received 3 new medicines in the rare disease programme. We appeal that the tender procedures are shortened (7 months is really an unexpectedly long period of time for a tender procedure). The resolution of the lack of medicines for rare and other diseases with emergency tender procedures is not the long term solution we desire. The lack of medicines and expecting procurement is an additional stress for both patients and families . We expect that medicines are procured for Spinal Muscular Atrophy as promised and oral therapy for Gaucher patients. Additionally, medicines are needed for pulmonary fibrosis, tablets Fludrocortison are needed for congenital adrenal hyperplasia, for Duchenne and Becker muscular dystrophy and others. There always new diagnosis and there is also research and new medicines are being approved in the EU and USA .
From the HIFM we are still waiting for approval for reimbursement for special bandages and band aids for Epidermolisis Bulosa these were promised back in 2016 in February. Also we hope that HIFM will have understanding to put breathing machines on the list of orthopedic aids (for patients who experience breathing aparatus failure and who cannot breathe independently).
Of course, the priority remains for diagnosis, for which still there isn’t reimbursement from HIFM or from the Ministry of Health and we expect that this will be remedied soon. In communication with MASA and the MH we hope that the financial obstacles are soon overcome for receiving diagnosis in Macedonia and abroad.
We are grateful to everyone who has supported us so far and we continue the cooperation in future . We hope that as an association we will contribute towards the future of people with rare diseases so that is the future that they desire.