A 2-year-old warrior from Pszczyna is battling against spinal muscular atrophy. An extremely expensive gene therapy is the only hope for the boy. It costs over 9 million zlotys (over 2 million dollars). Anyone can help by donating money to the fundraiser account for Maciuś Cieślik, but also by taking part in various activities organized by good-hearted people.
Family, friends, relatives and, above all, mum Ewelina her son are fighting for “the world’s most expensive medicine”. Without the drug the future is uncertain for little Maciuś. Starting the fundraiser is not the only way to collect money for the therapy. Dozens of hours on the phone, contacting a huge number of people, initiating actions across the country and abroad – all put us on a path to success.
It’s the most popular form of support – you take part in an auction as a buyer or seller. The full sum from the transaction goes directly to Maciek’s fundraiser account!
All kinds of social initiatives, such as engaging well-known people, contacting the media including the press and TV, promoting the actions in social media, or organizing charity events.
All kinds of events such as concerts, rallies, picnics, sporting events, and other charity gatherings aimed at collecting funds for Maciek.
It is a severe rare disease which, due to a genetic defect, causes the loss of spinal cord neurons responsible for the contraction and relaxation of muscles. The lack of nerve impulses leads to generalized weakness and progressive atrophy of skeletal muscles, and ultimately partial or total paralysis.
The disease affects people of different ages, but in more than 90% of cases the symptoms appear in infancy or early childhood.
The disease is diagnosed at some point in the life of one in 8,000 people, with about 80% of all cases being diagnosed in infancy or in early childhood.
According to the information from genetic laboratories in Poland, this is the estimated number of SMA cases diagnosed in Poland every year.
According to a study conducted by Dr Jędrzejowska, MD, PhD, approximately one in 35 people in Poland is a carrier of the SMN1 gene mutation causing spinal muscular atrophy.
This is the number of people listed in the Polish Registry of SMA Patients as of 1 January 2020. It is estimated that across the country 800-1,000 people suffer from spinal muscular atrophy.