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Press Release – 28.02.2013. – Rare Disease Day

Press Release

Rare Disease Day
28th of February, 2013th

The Association of citizens for rare diseases “Life With Challenges” marked the Rare Disease Day, 28th of February and with that it started the campaign for raising public awareness for rare diseases in Macedonia.

This year, except the patients and families that cope with Gaucher, Phenylketonuria, and Alagille Syndrome, towards the association joined patients and families that cope with Hereditary Angioedema (HAE), Morbus Leigh, Epidermolysis Bullosa, Congenital cataract, Tyrosinemia etc.

The president of the association, Vesna Stojmirova, emphasized that the association is opened for all patients and families that are challenged in their lives with rare diseases, and together they should try to step up towards a better future and a higher quality of life.

“Today we are here to support families that live with rare diseases and to revive the hope that the rights of the patients (treatment) will be fulfilled in Macedonia. For no, the condition of treatment is not changed compared to the previous year. Two patients with mucopolysaccharidosis are treated from the Ministry of Health in Macedonia, four patients with Gaucher are being treated by donation from Genzyme (for 4-6 years), one patient with Gaucher is on clinical study through Genzyme. In negotiation with the Ministry of Health in Macedonia we came to reforming of the Commission for Rare Diseases in Macedonia and the chairman of this commission, Aspazija Sofijanova (director of the Children Clinic in Skopje) is today with us. We hope that with the work of the commission we will increase the cooperation with the Ministry for Health in order to find long-term solution for provision of treatment for the patients with rare diseases. Also for some medicines that are available for other diagnosis from the Health Fund in Macedonia, we hope that they will be available for patients with rare diseases that need such medicines in the everyday health care process.”

At the event Mrs. Mary Jo Wohlers, wife of the ambassador of America in Macedonia, was present to support the association and the patients in their efforts as an honorary member of the association.

“If we work together, we will find solutions to the problems that patients face because of rare diseases. The work of the association is very meaningful and alsto the cooperation oof patients, families, and state institutions is a key factor to provide long-term solution for the problems of the patients with rare diseases.”

During the event, the director of the Children Clinic in Skopje, and the chairman of the Commission for Rare Diseases – D-r Aspazija Sofijanova, shared a good news, that one child with Gaucher will start treatment with the help of the Ministry for Health.

“With the establishment of the Commission for Rare Diseases we made a step forward towards supporting the patients with rare diseases, and providing treatment for normal growth and development of the patients. We are on a very good way to accomplish our goals. We can do everything if we believe. “

Mrs. Tanya Colin – Histead, director of the European Gaucher Alliance, emphasized that as a mother of a child with rare disease (Gaucher), she has even stronger motive to support the fight for treatment of the patients in Macedonia.

“Even though we are European Gaucher Alliance, we work through the whole world. Until know we have accomplished a lot, and especially the last years we succeed to move forward regarding the progress of treatment in the neighbor countries of Macedonia. Raising awareness about rare diseases in Macedonia and finding the way for treatment of the patients with rare diseases is also my personal challenge and I hope that we will find solution for treatment of the patients.”

The Association of citizens for rare diseases “Life With Challenges”, will continue with the campaign for raising public awareness about rare diseases in Macedonia and with the efforts for provision of treatment for the patients with rare diseases in Macedonia.

A lot of families that face rare diseases were present at the event for Rare Disease Day, joining together in the efforts for providing a better life for their children.

*The event was supported by: Genzyme, with the PAL award, CONCEPT Marketing and PR, the Medical Faculty, the Children Clinic in Skopje, City Mall and Pelisterka. Students from the Faculty of Pharmacy were volunteering in the organization of the event.

cooperation between HAE Macedonia and Life With Challenges

„HAE Macedonia“ is a patient association for Hereditary Angioedema, and it is founded in 2009. In Macedonia there are around 10 patients diagnosed with this disease, and together they try to cope with the physical and psychical consequences. It is hard to be a parent but it is even more difficult when your child has a rare disease. HAE Macedonia is trying to provide support through different activities, discussions and similar.

HAE MAcedonia started cooperating with Life With Challenges, in 2012. Together patients, parents and supporters are trying to accomplish the mission  – providing quality life for patients with rare diseases.

We hope that our cooperation will continue as fruitful as it started with the campaign for raising public awareness about rare diseases. hae logo

Annual activity report – 2012

Association of citizens for rare diseases LIFE WITH CHALLENGES – Bitola

The Association of citizens for rare diseases LIFE WITH CHALLENGES – Bitola is founded by patients and parents that face life with Gaucher disease in the year 2009. Gaucher is a disease that comes from lack of enzyme in the organism and there are problems because of storage of Gaucher cells in the liver, the spleen and the bone marrow. Because of that these patients have low hemoglobin levels, enlarged spleen and liver,weak bones, often fractures, lost of energy, fatigue etc.

Today in Macedonia there are 13 patients diagnosed with Gaucher disease, 4 of them are on Cerezyme donation bu the ICAP program and 1 of them is on clinical study on Eliglustat. They are receiving therapy for 3-4 years and all the symptoms are almost totally gone.  This therapy is a life therapy and patients should receive it every two weeks through infusion.

Later in 2012, the association started growing, more patients and parents joined with other rare diseases. First parents with children with Phenylketonura joined. This diseases causes brain damage and mental retardation, associated with physical difficulties. In Macedonia there are 7 patients with this disease and they provide food and medicine on their own wo they can provide normal life.

With Alagille Syndrome there is one child diagnosed in Macedonia. This genetic disorder is characterized with progressive loss of the liver function and in 30 to 50% of the cases there is cirrhosis, yellow skin, itching, problems with vision. To improve their condition and maintain normal development and growth this child needs treatment for the liver, the skin and the eyes and special diet.

In the association there are also patients with HAE, rare disease manifested by swelling (edemas) on different body parts. There is no cure, but there is treatment with C1 Inhibitor. In Macedonia there are around 16 patients with HAE.

There are other rare diseases in Macedonia as: multiple myeloma, phenylketonuria, myelodysplastic syndrome, gastrointestinal stromal tumors, recidivans, acromegaly, gastroenteropancreatic neuroendoc rine tumors, mucopolysaccaharidosis, hereditary tyrosinemia, galacorosemia, and hereditary fructose intolerance.

The association is open for members and other patients and parents that face life with rare diseases. In Macedonia there is a low level of diagnosis of rare diseases and we need to improve the area of diagnosis and treatment further. Patients often go in other countries for treatment and diagnosis.

Until now the Ministry of Health is treating only 2 patients with mucopolysaccaharidosis.

Activities and events in 2012:

• Continuous activity form 2009 until today is regular communication with the Ministry of Health for improvement of conditions for treatment of patients with rare diseases.

•  Continuous cooperation with Children Clinic Skopje and realization of meetings with the director of the Chidlren Clinic – Aspazija Sofijanova, who become a supporter of the association towards realization of our goals.

• In 2012, on 29th of February, Rare Disase Day, with a goal of raising public awareness and informing of citizens and patients, we organized a press conference with media and we talked publicly about our probelms and challenges. more information about the event you can read on the links bellow from media.
9. Telma television – news-
11. novinska agencija –
13. Focus daily newspaper
14. Sitel television – morning show and news
15. Alfa television – news, Portik and morning show

• Cooperation with associations and organizations: HAE Macedonia, HEMOLOG, Associations form Bulgaria, Serbia, Croatia, Slovenia, Bosnia and Herzegovina etc.
On EU level we are members of European Gaucher Alliance and now in 2013th we are also full member of EURORDIS.

• Projects: In 2012 we won the PAL award and we will use the resources in 2013th according to planned activities for campaign for raising public awareness for rare diseases (press conferences, events, publishing info material).

• Web page: , We have a new web page where we publish activities and information. We continue with communication through facebook group and fan page: , .

• Honorary member: Ms Mary Jo Wohlers, wife of ambassador of USA in Macedonia.

• Companies: cooperation with Genzyme (Sanofi Company) continues for raising public awareness about rare diseases. CONCEPT Marketing Communications is also a supporter of the association in the field of public relations, design, promotion materials etc.

• Media: media cooperation is on a very high level. All of them publish stories, articles and interviews about rare diseases and some of them filmed TV shows on the subject.

Future activities:

During our development we focused on contacting patients, uniting families and offering support and help in terms of information and treatment. As the membership is growing the association becomes stronger and more influential.

We are also making efforts in creating a register for patients with rare diseases so we can see the real situation in Macedonia regarding rare diseases.

Other activities are planned in cooperation and communication with the members and supporters of the association. We expect to come to long-term solution for provision of treatment for rare diseases in Macedonia in communication and cooperation with the Commission for rare diseases, the Ministry of Health, the Health Insurance Fund of Macedonia, the Bureau of Drugs and the Children Clinic. .

Vesna Aleksovska
President od the Association of citizens for rare diseases

Rare disease day – 28th of February

On 28th February we will organize a media club/round table with journalists, representatives from the rare disease association “Life With Challenges”, doctor and representatives from the Health Institutions in Macedonia. The association will present its goals and explain the problems/issues of the patients with rare diseases. Further in an interactive discussion the doctors will answer questions from the present audience and they will advice about improving quality of life of the patients.

This event has a goal to raise public awareness and open a dialog between the interested parties. Patients and journalists will have a possibility to talk about the challenges they face every day and about the further activities of the association.

More details about the event … soon …

Balkan regional meeting of Gaucher associations

Dear members and supporters,

From 5th to 7th of April, in Sarajevo, Bosnia and Herzegovina, there will be a Gaucher meeting organized by the Balkan Associations from Slovenia, Macedonia, Bosnia and Herzegovina and Serbia.

The goal of the meeting is for patients, parents, doctors and nurses to talk together about life with Gaucher and how to improve quality of life in the future.

At the same time, this is a first meeting of patients with lysosomal diseases at Balkan region level.

The interactive program is on the following subjects:
– Mutual understanding and support

– Exchange of opinions and experience

– Questions and discussion about life with Gaucher

The meeting will be in hotel Europa in the center of Sarajevo and as official language we chose Serbian-Croatian with a goal to understand each other easier and better and to establish and maintain contact with each other.

This meeting is organized from the following patient organizations:
Association of patients for rare diseases from Bosnia and Herzegovina  –
Association of citizens for rare diseases “Life With Challenges” – R. Macedonia–
Association of citizens for help of patients with Gaucher  Serbia  –
Association of patients with Gaucher – Slovenia  –

Logistic and financial support for organization of the patient meeting is provided by:
Genzyme (Sanofi Company)
Association XY –

Vesna Aleksovska
President of Association of citizens for rare diseases “Life With Challenges”

PAL Award – Patient Advocacy Leadership Award

The Association of Citizens for Rare diseases “Life With Challenges” from Bitola is a winner of the  PAL (Patient Advocacy Leadership).  As winner of this award are 8 more organization from the world.

The PAL Award recognizes patient advocacy organizations for extraordinary work and innovation in helping to raise awareness of lysosomal storage disorders (LSDs), a family of rare diseases that often affect only a small number of people around the world. LSDs typically cause progressive and debilitating health problems (

Fifty patient organizations from around the world applied for 2012 PAL Awards by submitting proposals for innovative campaigns and projects to support the LSD patient community.

Proposals were submitted from organizations that represent people affected by Gaucher disease, MPS disorders, Fabry disease, Pompe disease and Niemann-Pick disease, among other rare diseases. Applicants came from six continents, and 2012 winners were recognized in Chile, France, Hong Kong, Macedonia, Poland, Thailand, United Kingdom and the United States.

“For 3 years, “Life With Challenges” has been working to help people learn about and cope with Gaucher and other rare diseases. The PAL Award will help us to continue to build on these important efforts in the years ahead. We are very excited about the opportunity to expand our work and our impact through this important honor,” said Vesna Aleksovska, President of “Life With Challenges”.

According to the Global Genes Project, an estimated 350 million people worldwide are affected by rare disease. Only five percent of rare diseases currently have approved treatments and more than 50 percent of rare diseases do not have foundations, advocacy groups or organized community support to help expand awareness and improve access to care.
“The organizations that were selected to receive PAL Awards for 2012 showed a remarkable level of innovation and dedication in helping others to learn about and cope with lysosomal storage disorders. Genzyme is honored and very pleased to be able to support the work of “Life With Challenges” through the PAL Award program this year,” said Vulnet Ferrati, Genzyme representative from Macedonia.


Press – media announcements

Press from 29.02.2012 – Rare disease day – press conference

Daily newspapers

  5. Focus – on line link not available

National televisions

  3. News at 18.30h on Telma-
  4. Sitel – News at 15.00h- on line link not available
  5. Sitel – morning show – 8.30h – on line link not available
  6. Alfa – portik – tv show – 10.00-12.00 h. – on line link not available
  7. Local television from Tetovo – on line link not available

Новинска агенција / News agency


Internet portals


Press information

The government is ignoring 20 patients with rare diseases
With the budget cuts, the hope of the patients is gone!

A 12 year old girl with Gaucher will have to be operated if the Ministry of health does not make an intervene import of the needed medicine. The budget for the program with rare diseases has been cut during the last budget rebalance and there is no budget for treatment of the patients with rare diseases in Macedonia.

Mence Atansasova – Toci

The medicine exists but is not available for the 12 year old A.K. who has Gaucher. She will have to be operated because her condition is getting worse. Her spleen is enormously enlarged and her liver also. That is why the spleen has to be removed. But if she starts enzyme replacement therapy as soon as possible, she won’t need the operation. The girl was diagnosed 6 month ago and the parents noticed the signs when her abdomen was getting bigger because of the enlargement of the spleen and liver. The necessary therapy should be provided by the Ministry of Health. Her family is desperate, trying to provide the needed medicine. They contacted the Ministry but there is still no answer.

– My daughter has constant pain in her abdomen. The liver and the spleen are enlarged and she has to start treatment immediately. Otherwise her spleen has to be removed and that is not good. – says her father Shani K.

The medicine is expensive and it is not available in Macedonia. The company that produces the medicine is ready to provide the first 50 units of the medicine and they will wait for payment for a year if needed (if the government is not able to pay sooner). The association “Life with Challenges” is negotiating with the Ministry of Health to provide the needed medicine and save the life of the girl.

– We hope that the Ministry of Health will bring the right decision – providing the needed medicine. It is about saving human lives that we are talking and we need long-term solution for the patients with rare diseases in R. Macedonia. – says Vesna Stojmirova, the chairman of the association.

The newspaper “Den” asked the Ministry of Health for an answer about the pending issues regarding the condition of the patients with rare diseases, but there was no answer from the Ministry. It is still not known if the girl will have a normal life or a life with lower quality and psychical and physical obstacles.

In Macedonia there are around 20 patients with rare diseases and most of them are children. In the program for rare diseases it is stated that 1 million Euros will be provided for the treatment of these patients. With the last rebalance of the budget this program was ignored and with the budget cuts, the hope of the patients is lost. From the association “Life with Challenges” they say that through the program only 2 children with the rare disease – mucopolysaharidosis are treated and all the other patients are left without therapy.

The doctors say that if these patients are treated they can have normal lives. Without treatment they are facing serious health problems and most of them die at young age. The treatment for these patients will cost approximately 500 000 Euros. The company that produces the medicine for Gaucher is already donating treatment for 5 patients with Gaucher in Macedonia. In the past 4 years they donated around 5 million Euros for treatment and diagnosis. They are committed to treating the patients and that is a huge help for the government in Macedonia.

For raising public awareness about rare diseases in Macedonia, the association “Life with Challenges” will organize a campaign with the resources from the PAL reward (Patient Advocacy Leadership). For the PAL award, 50 countries in the world submitted applications and only 9 of them received the reward. The project “Do not cry fight for your rights!” will be a huge support in the fight for patients’ rights in Macedonia.

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