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A volunteer of LIFE WITH CHALLENGES Gordana Loleska, in cooperation with companies and organizatins from Ohrid managed to organize teams who run in support of people with rare diseases in the event Ohrid Running.

This was a promotional event with a main goal to support running, sport, quality of life and healthy life.

Statement Gordana Loleska, member of LIFE WITH CHALLENGES

„For the children with rare diseases and special needs on this event we had professionals but also parents and patients involved. The citiznes also supported our idea for raising public awareness on rare diseases. People with rare diseases deserve better quality of life, they deserve a chance to do most with what they have. Everyone can be successful if he has a chance to do so.“

Statement Vesna Aleksovska, president of LIFE WITH CHALLENGESИ

„Gordana is one of our most active members, she organizes many events in Ohrid and we are proud of her. This is just one more event showing support for people facing life with a rare disease. We need to remember that there are many social and health services needed to support families with rare diseases.  

After the register for rare diseases started in 2015, there are around 450 patients registered and around 40 basic diagnosis (70 different). With the new financing of the program for rare diseases at the Ministry of Health, now there are around 29 medicines for 15 around 15 different diagnosis in Macedonia. There is a need for more medicines for different diagnosis. If there is a drug, patients need to have it so they can have hope for life. We expect that medicines will be covered soon for FAP, Spinal Muscular Atrophy, Muscular Dystrophy – Duchene, Myelodisplastic Syndrome – MDS, Acute Myeloid Leukemia, Pulmonary Fibrosis and others.  We also ask for social services such as personal assistants for ALS patients and other patients with severe physical invalidity. We are still waiting on the Health Insurance Fund to approve refunding for special bandages and plasters for Epidermolisis Bulossa and other things that patients need.

We hope that the new Ministers of Health and Social Policy and Labor will find a way to make the Program for Rare diseases better. Institutions need to work together to make life easier on families with rare diseases.“

The event was supported by:

Tamaro Markets, Youth Club Red Cross Ohrid, NGO Option Ohrid, Cuba Libre IOhrid, Poposki Ordination Ohrid, Ohrid Super star talent show

Know AML is the world’s first global AML awareness coalition supporting AML World Awareness Day on April 21. The mission of Know AML is to raise awareness and education in AML to patients, carers, families, healthcare professionals and the general public.

In December 2016, during the American Society of Hematology (ASH) meeting, a collective of AML patient Leaders, professional and industry representatives, now named the Know AML Coalition, gathered to agree on how to mark AML World Awareness Day in 2017.

The Fight AML campaign aims to improve education and support in order to empower the AML community in its fight against AML and it is the first of many Know AML awareness and education activities.

More info on – http://www.know-aml.com

The Life With Challenges association, together with the National Alliance for Rare Diseases of the Republic of Macedonia, under the umbrella of the European Association for Rare Diseases – EURORDIS, celebrated the international Rare Diseases Day, February 28, 2017 this year as well under the motto “With Research, Possibilities are Limitless”. In addition, this year we are also focusing on parents of children with rare diseases and the difficulties and challenges they face. Their stories were on display in a poster presentation in the hall of the EU Info Center today and they were shared on social media.
After the new Law on Financing the Rare Diseases Program was passed in 2015, 21 new medicines were procured for approximately 12 rare disease diagnoses, which covered more than 150 patients. According to information available from the Ministry of Health’s public bids, the number of medicines in 2016 has increased by 29, which means that more diagnoses have been covered, so now the number of patients receiving medication has reached approximately 220. There are 46 primary diagnosis in the registry.
Statement by professor Aspazija Sofijanova, PhD, president of the National Commission for Rare Diseases:
As medical doctors we must be less emotional, however that’s not so in our case because we are in direct contact with sick children. When we save a child, it feels like we have saved the world, but when we lose a child, it feels like we have lost the world. This year we will continue to struggle for the patients with rare diseases, and the parents, the patients and the associations offer the greatest help in that struggle – if we join forces with the institutions, there is a lot that can be done. We know the issues. I just came from the clinic and I would like to say that Iskra says hi, that’s a child who has spinal muscular atrophy and who is still in intensive care at the clinic. I also know Daniel, who was helped a great deal by the US Embassy in Macedonia, that is Mrs. Mary Jo Wohlers. It is a new medicine, and we will try to find the financial means to bring it to Macedonia, like we bring medicines for other patients.

Изјава, Проф. Д–р. Аспазија Софијанова, Претседателот на Националната комисија за ретки болести

Statement by academician Momir Polenakovic, Macedonian Academy of Sciences and Arts (MASA):
Within MASA we have a small genetics and diagnostics center, but we also collaborate with other countries and we are trying to forge the way ahead in terms of education and to push forward in diagnostics, which is as important as prevention. Early diagnostics is essential for early therapy and for providing timely information to the patients, so that their life quality is improved. The collaboration with the Wilson Disease Association – Macedonia has already resulted in improvement in the education for diagnosing Wilson Disease as well, and they provided us with reagents through a donation. We hope that in the future we will have more funds for providing other reagents in order to boost the diagnosis of a larger number of rare diseases.

Statement by Rashela Mizrahi PhD, Ministry of Health of the Republic of Macedonia:
The rare diseases program was launched two years ago with the creation of the rare diseases registry and the increased financial support for medicine procurement. There are many issues in terms of diagnosis, classification and systematization. There’s a whole apparatus working in the background at the Ministry and the Rare Diseases Commission, where clinicians work pro bono for the wellbeing of the patients. We have also established criteria and requirements for entering the registry, some 400 patients have been registered with more than 46 diagnoses, and we already have medication for 220 patients.
Statement by Rebeka Jankovska Risteski, president of NARBM (National Alliance for Rare Diseases of the Republic of Macedonia):
This year we are focusing on patients who are yet to receive medicine. Such are patients who have spinal muscular atrophy, children whose only hope is a medicine approved by the American Food and Drugs Administration last year. Then we have the medicines for patients who have rare forms of cancer, such as the MDS patients – two patients who were donated the medicines have already shown signs of improvement in their wellbeing and life. Therefore we believe that innovative medication should be made available in order to allow for a good quality life for the patients and their families. Then we have the medicine for juvenile arthritis – only one of the three that exist is being procured. Finally we would ask the Health and Pension Insurance Fund of Macedonia (FZOM) not to forget that even in 2015-2016 they promised the patients suffering from Chrohn’s disease and ulcerative colitis they would provide them REMICADE. Of course, we would also like them to provide patients with medical aids and meet other needs when there are no medicines available. Our vision as an alliance is for nobody to be left behind.

Statement by Anja Bosilkova Antovska, vice-president of NARBM:
There is a great variety of rare diseases and anyone may contract them. We carried out research in collaboration with the Wilson Disease Association – Macedonia and we drafted some recommendations for changes in Macedonian legislation so that rare disease patients are not apprehensive of whether there will have a long-term therapy plan available or not. A national rare disease program and systematic, legal solutions must exist for us to ensure a future for the rare disease families.
Statement by Vesna Aleksovska, director of Life With Challenges:
No matter how much I speak I am bound to leave something out. On the one hand we are thankful for the understanding that the institutions have shown for us, rare disease patients, in the last two years, but on the other hand I must also say that there is a lot to be done, many people to be helped, and that we will always have new requests and new needs. The rare diseases are very unique, each one has a number of variations and each patient is a different story, with separate challenges, difficulties, issues, happiness and sorrow. Working together as part of the alliance I think that we can achieve much more, and I hope that we will be increasingly vocal and that our voice will be heard more and more. Each family deserves the best chance possible for a happy life. We will continue to meet up with the institutions, we will speak up in front of the media, we will get the public involved by organizing events… Each patient must be taken care of.

Other events around Macedonia

Bitola
This year the rare diseases awareness raising campaign began with an event in Bitola on February 24, at 10 a.m. in downtown Bitola, on Sirok Sokak, where with the support of the Hepar Centre for Citizens who have Rare Diseases the Life With Challenges Bitola organized a press-conference and distributed flyers downtown in order to raise public awareness for the rare diseases.

Ohrid

On February 28, 2017, at noon, a march began from the front yard of the Hristo Uzunov primary school. Some 200 students in a line marched along Pitu Guli street, and then along Jane Sandanski street, and then they joined forces with the students of the St. Clement of Ohrid secondary school and they continued together all the way to the Macedonia quay (in front of the Royal hotel). There, at 1 p.m., the students ran a cross race to the Ss. Cyril and Methodius monument. From there the students went to the city square, where they distributed information leaflets for rare diseases. On February 26 and 27, there were hiking activities organized as well, with the support of ZSH Avantura – Ohrid. The activities in Ohrid were also bolstered by the Animalija association, which supported the idea for rare disease awareness raising with the motto: “Adopt a pet, for some people that’s the only free therapy”. The event was organized by Gordana Loleska, a mother of a child suffering from Alport syndrome, with the support of the Ohrid Red Cross, the St. Clement of Ohrid secondary school, ZSH Avantura, the Animalija association and the citizens of Ohrid.

Prilep

On February 24, 2017, a group that distributed flyers for rare disease awareness raising joined the Prilep masquerade. There was also a public lecture for rare diseases. The event was organized by Jasmina Hristoska, an acromegaly patient, with the support of SLAP – Prilep.

Last year a member of the association for rare diseases Life with challenges, a mother with a child with Alport syndrome, Gordana Loleska, employed in the post office in Ohrid, started an initiative for creating a postal stamp with the logo of rare disease day to help in raising awareness for rare diseases in Macedonia. This initiative was supported by many medical professionals in Macedonia who send letter of support to the Direction for philately to the Director Mr. Elez Elezi.
We did an internet search and we realized that this kind of stamp will be first of its kind – dedicated to Rare Disease Day to all the Children with Rare Diseases and it is a great initiative for the Macedonian post office, an accomplishment on world level. This year the post accepted this initiative and we hope that the stamp will be ready for distribution for rare disease day 2017. The name of the stamp will be Children with rare disease.

The stamp will be promoted by the national alliance fir rare diseases of Macedonia and also by the post office. It is a great idea that came true … a nice way to promote awareness.. we hope that other countries will do this also.

We are very proud of Gordana Loleska who enthusiastically took this idea forward. We also want to say thank you to the general director of the post office Mr. Fadis Redzepi, to the director of the sector for philately Mr. Elez Elezi, to the director of the post office in Ohrid Mr. Sejfula Hani and to the council for philately at the post office of R. Macedonia, with its president Ms. Vasilevska Sonja.

For the 15th time, the members of the Dicing centre Amfora, and the Club for under water activities Ohrid, in cooperation with the Alliance for under water activities of Macedonia, decorated the Christmas tree under water in Ohrid lake, Macedonia. Traditionally as every year they decorated the tree in the lake at the Bay of Bones. Even the youngest members of the diving club participated in this activity.

This year the Ohrid divers dedicated the decoration to the children with rare diseases in Macedonia. Goran Balevski said that this year they decided to sent a message to the public for support of the children with rare diseases.
This cooperation was made possible with the efforts of our member Gordana Loleska from Ohrid.

On 3rd of December, our member Viktorija Penovska with the help of her school and her friends and family organized an event in her school focusing on the importance on accepting disability and the importance of social inclusion.

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The Macedonian Academy for science and art had the opportunity to organize the fifth meeting of SEE for rare diseases, on 12th November, 2016.
Int he organizational committee of this meeting are: Momir Polenakovic, President, Zoran Gucev, Secretary, Nada Pop-Jordanova, Ilija Filipche, Zivko Popov, Velibor Tasic,Aspazija Sofijanova, Vesna Aleksovska, Dijana Plaseska Karanfilska

The program was as it follows:
09.15-09.30 Stefan Chichevaliev, Vesna Aleksovska, Skopje Macedonia, Policies, practices and their impact on the quality of life of people with rare diseases
09.30–09.50 Momir Polenakovic, Skopje, Macedonia, Rare genetic kidney diseases
09.50-10.15 Milosevic Danko, Zagreb, Croatia, Atypical hemolytic-uremic syndrome (aHUS) in Croatian children
10.15-10.40 Zoltan Prohaszka, Hungary, Overview of complement deficiencies
10.40-11.10 Milan Lakocevic, Belgrade, Serbia, Enzyme Repleacement treatment: Serbian experience with taliglucerase alfa
11.10-11.40 Adrijan Sarajlija, Belgrade, Serbia, Glycogen storage diseases – single center experience
11.55-12.20 Johannes A. Mayr, Salzburg, Austria, Mitochondrial diseases – Are they rare or common?
12.20-12.45 Velibor Tasic, Skopje, Macedonia, Pitt Hopkins syndrome an update
12.45-13.10 Zoran Gucev, Skopje, Macedonia, The spectrum of growth disorders: the Macedonian part
13.10-13.35 Rossella Parini, Milan, Italy, Mucopolisacharidosis: diagnosis and treatments
Session II – Moderators: Ilija Filipce, Velibor Tasic, Zivko Popov
14.10-14.40 Hans Joachim Seitz, Hamburg, Germany, Endocrine Disruption as cause of (rare) diseases
14.40-15.10 Oliver Bartsch, Mainz, Germany, Next generation sequencing in pediatrics (in Germany)
15.10-15.35 Jan Halbritter, Leipzig, Germany, Mutation analysis in kidney stone disease
15.50-16.05 Zlate Stojanovski, Skopje, Macedonia, Polycythemia rubra vera in patient with Gaucher disease
16.05-16.25 Liljana Saranac, Nish, Serbia, Microprolactinoma in childhood and adolescence; a rare or underestimated disease
16.25-16.45 Zoran Gucev Skopje, Macedonia, M.Gaucher: treatment

The Citizen Association for Rare Diseases “Life with Challenges” organized a round table on the topic of rare cancers in Macedonia at Best Western Hotel in Skopje on October, 28th , 2016. This activity was about raising the institutional and public awareness regarding the inaccessibility to treatment for patients suffering from rare forms of cancer for which at the moment no therapy is procured neither through the FHIM nor through the rare disease programme of the Ministry of Health nor through the oncology and haematology clinics.

One of these rare cancers is the Myelodysplastic Syndrome and the day of MDS is marked in the month of October. One MDS patient is already receiving treatment through donation and another one received a similar donation in order to stabilize the state before starting bone marrow transplantation. There are different types of MDS and they fall into the rare disease group and there are the so called orphan drugs for their treatment. Unfortunately, at the moment we do not have precise information about the number of patients in need of treatment because of the different classification of MDS, however, the probability is that around ten patients need innovative therapy which will suppress the disease progress and improve patients’ lives.

Also there are many other different types of rare cancers such as: multiple myeloma, malign myeloma, gastrointestinal stromal tumors, gastroenteropancreatic neuroendocrine tumors, acute myeloma leukemia, angiosarcoma, neuroblastoma and many others; some of these have been publicized as rare in the Rare Disease Programme back in 2009, yet today they are not included in the Rare Disease Programme. We believe that they should not be discriminated as such and that these patients should receive the necessary treatment as they in fact do so in our neighbouring countries such as: Bulgaria, Croatia, Slovenia, Montenegro and Serbia made progress in this area.

Statement Vesna Aleksovska, President of “Life with Challenges”

“Rare diseases were invisible for a very long time in Macedonia. Since 2015 and the commencement of the Rare Disease Registry and the increased finances in the Rare Disease programme finally offer 13 diagnosis, that is 150 patients have received treatment. Until then these patients did not receive any type of medication and were left to their own means. Unfortunately, the patients with rare forms of cancer who are at the haematology clinic and oncology clinic still do not receive the necessary medicines. As a patients’ association we believe that there is discrimination towards these patients because they are faced with a rare disease and should be provided with medicines that will prolong their life, as well as improve their quality of life. One of these rare cancers is myelodysplastic syndrome (MDS) which is marked in October and for which we have created a brochure aimed at patients facing with this disease. One patient from Macedonia with MDS already receives the necessary treatment through a donation. We believe that treatment should be provided for all patients with rare cancers who are registered at the clinics. Unfortunately we do not have the exact number at the moment, but such diagnosis are rare and can be found at the Orphanet list; and at the moment this is one of the criteria for procurement of medicines for rare diseases. The criteria of 20 patients per diagnosis is discriminatory and as an Association and Alliance for rare diseases we reacted to the number 20 because we fear that the number is very limiting and there is uncertainty what will happen when the 21st patient is registered. We appeal for the rare disease programme to additionally expand financially, that is to say, for the amount that had been promised at the beginning (twice more than the present amount of 3 million euros) so that more patients receive the necessary medicines. Innovative medicines used in the world should be used in Macedonia in order to prolong and improve the patients’ lives.

Statement by patient with MDS –Dragi Lazevski

“In September I felt as if without energy or fitness, after all the tests the diagnosis of megaloblastic anaemia was confirmed in November. I was in dire financial situation and unable to provide the necessary medicines. Fortunately through Doctor Irina from haematology, whom I am deeply grateful for all her endeavours and those of the nurses and staff at the haematology clinic, I received treatment through donation from the company Celgene. After the treatment I felt wonderful and I can work and spend time with my family and do everything that I did before the disease. I must say that is important that innovative therapy should be available to al patients in order to have good quality of life and to prolong their life. No family alone can afford to provide treatment. Financially it is terribly difficult.”

Statement – Biba Dodeva, President APO

“In this pre-election times citizens need more than slogans. Therefore I publicly call upon all public institutions, present and future government, present and future health managers, I call upon the politicians and all those which our health depends on to put cancer as a priority in the health programmes and to understand the necessity of URGENT implementation of the National Strategy for Controlling Cancer in Macedonia that will enable and engender joint detection of problems and a comprehensive approach for surpassing them and resolving them.

Statement- Davor Duboka, Executive Director of the National Rare Diseases Organisation of Serbia

“I would like to point out the importance of what has been happening in Serbia recently, namely the HIF is already in negotiations with the pharmaceutical companies to create a priority list of innovative medicines that will be procured through a special fund in order to help patients facing difficult diseases, not only rare disease, but also cancer and other diseases requiring innovative therapies. In Serbia all the associations have come together in our demands for innovative medicines and we have finally reached and understanding from the institutions for the needs of the patients. For the time being 20 new medicines will be procured through the list of priority innovative medicines.”

Statement – Borislav Gjuric, President of the Rare Disease Association in Bosni and Herzegovina

“Bosnia and Herzegovina are unfortunately in a bad political situation because of all the problems we were facing in the past and have been facing ever since. The separation into cantons is very complicated and in some regions the situation with medicines is very good while in others it is devastating. Macedonia has a simpler political system and it is really incomprehensible why there is not a solution for the cancer patients yet. Personally I think it is literally manslaughter not to provide medicines for a patient whose life could be spared. It is not manslaughter just to kill someone. Health is a basic human right and no citizen should be deprived of it. All countries in the region make efforts to improve the quality of life of patients; we collaborate with all the associations and support regionally each other and we will not give up from the basic human right of life – health. We cooperate with institutions and media as well… one by one we might be few, but together we are many and together our voice is stronger and reaches further.”

To mark the International Gaucher Day, the Macedonian association for rare diseases LIFE WITH CHALLENGES together with the Albanian association organized a meeting on 8-9 October in Struga, Macedonia. At the meeting we had families, patients, doctors and nurses connected to Gaucher disease type 1,2 and 3. Also we had representatives from Genzyme and Pfizer who sponsored this meeting.

Patients talked about their challenges and problems regarding therapy and they shared experiences about what is going on in their countries at the moment, how they were diagnosed, what kind of therapy do they receive, and what kind of tests do they do … they talked about home treatment and will it be a good option to have in future. We have the possibility to also hear a story from a patient who was on a clinical trial on Cerdelga and now he is still on the pill, which was a new experience for all patients. We truly hope for a future where patients together with doctors can choose therapy that is most suitable for the patient and with most quality of life for the patient and the family.

Doctors from both countries (Dr Biljana Coneska Jovanova) had presentations about what is Gaucher disease, and about the patients in the countries (treatment, challenges, future options).
We realized that we face some same and some different problems and that we can help each other to do more for the patients.

The support from EGA is very important, because of EGA we manage to came together and talk, and now there is an association of patients in Albania that will soon join EGA. We agreed that the most important thing in future that we expect from EGA in this region is education of doctors about diagnosis and treatment. We hope to have the next meeting in Albania in spring time and to have guest lecturers – doctors from EU.

The Citizens’ Association for Rare Diseases Life with Challenges continues the tradition to participate in climbing Macedonia’s highest mountain top Golem Korab in 2016.

On the occasion of 8^th of September the traditional climbing of Golem Korab takes place on the 4^th of September, Sunday. ZPK Korab pitched  the flag of the Citizens’ Association for Rare Diseases Life with Challenges on the mountain top.

Statement by Vesna Aleksovska, Chairperson of the Association

We are grateful that the rare disease programme with the Ministry of Health works with a greater budget and encompasses a larger number of diseases, that is to say, more medicines are procured for rare diseases and they literally save families. I sincerely thank the team within the Ministry of Health,  especially Rashela Mizrahi who heads the programme with great dedication and understanding of our challenges. A lot of people are responsible for the advancement in the field of rare diseased in Macedonia, firstly the patients and parents who courageously demand their rights from the media and the institutons. They have received selfless support from the doctors headed by Aspazija Sofijanova, MD, as the chairperson of the Committee for Rare Diseases.

We still face various challenges that we hope will be resolved in cooperation with the institutions. Firstly, it is very important that the tender procedures are realized swiftly and efficiently and patients should not be left to wait 3 to 6 months without treatment as was the case this year. We have understanding for administrative procedures and in return we demand understanding for the needs of patients. No one should be left waiting for something that means life for that person.

Furthermore there is the need to clearly establish how new patients will be registered as well as how new medicines will be introduced in the list in the programme in order to avoid confusion and wandering from one institution to another and again long waiting periods for the patients. These diseases are lifelong, chronical and there is the necessity of an urgent intervention so as to avoid long-term consequences to health and quality of life. Accordingly, if a condition requires combined treatment it should be timely procured and should the patient become resistant to the first-line therapy to provide second or third-line therapy if such therapy exists.

We appeal that it is of utmost importance to provide treatment for patients with rare cancers who do not receive any treatment whatsoever at the moment neither through the MHIF, nor through the cancer programme, nor through the rare disease programme. These cancers are very few and the number of patients is very low and it is necessary to act swiftly to resolve the issue with these patients. Their families suffer quietly looking for hope through donations and alternative treatments.

In case where there is no treatment available and there is a need of orthopedic devices they should also be provided through the MHIF and the reimbursement should be alleviated with a statement which would be issued twice a year and not each month. Some disabled patients have the need of a personal assistant and assistance after the age of 26 and we appeal for this to the Ministry of Labour and Social Policy. All institutions should work together to resolve all the issues and challenges which the families with rare diseases are faced with.

Where necessary patients should be provided with the opportunity to receive treatment at home or in a hospital in the cities where they live in order to lower the costs of both patients and clinics as well as the time necessary to access treatment.

Finally I have to point out the importance of carrying out the procurement through suitable distributive channels so that to avoid problems with the quality of medicines, that is to say, to have additional requirements when there is a case of parallel import of medicines where a certificate of quality and origin shall be requested. This should be so in order to avoid additional patient suffering as was the case with the patients with Gaucher who were left without treatment for 6 months and for which there is still no reply from the Agency of  Medicines which withdrew the medicines in order to investigate their quality after a strong allergic reaction with a child suffering from Gaucher.

This activity is carried out in order to raise the public awareness regarding the rare diseases in Macedonia and the challenges the people with rare diseases and their families   face daily. For this activity we are grateful to ZPK Korab and its chairperson Ljubomir Kotevski who accepted the idea wholeheartedly from the very beginning.

We are grateful to all those who have supported us so far and we continue the cooperation. We hope to make our contribution as an association to the future for the people with rare disease and to create a future that they desire.