Our main concern with creating this website is to make the disease NBIA-BPAN better known and to collect donations to fund research and to fight this severe neurodegenerative disease. Our BPAN families from the U.S. are very active in this respect and have already managed to fund two research fellowships, each amounting to $45.000,00. Such means are regularly distributed by the NBIA Disorders Association (NBIA DA) which is located in the U.S. The organisation cooperated with the German patient advocacy association Hoffnungsbaum e.V. in the research project TIRCON which was financed by the European Union from 2011 to 2015. The TIRCON consortium has set up a patient registry and a biobank for the research of all NBIA types, including BPAN, at the Friedrich Baur Institute at the LMU Munich. After the EU programme ended, they have been financed by the NBIA-Alliance, a cooperation of international NBIA patient advocacy oragnisations. Funds provided through donations for the research on BPAN enabled the NBIA DA in spring 2018 to tender two further research fellowships. The Italian partner organization AISNAF funded another three research projects on NBIA in July 2018, including one on BPAN. This will be the first research project on BPAN being financed by donations to Milly’s Mission.
Combating another NBIA disease (PKAN) has made progress so that a medication has been tested since 2013 in the framework of a study. Other PKAN medications are initially being tried out in clinical studies. Since this demonstrates that developing NBIA medication is realistic we are eagerly awaiting the results. The people affected by NBIA do not have a big lobby. In Germany, unfortunately, there are no state subsidies for researching rare diseases and the means of the EU to fund research in the longer term are not sufficient, either. That is why the families have to become active and to push forward research funding themselves.
The BPAN families’ short term goal is to finance the research and development of efficient therapies for BPAN patients in order to stop the neurodegenerative progression in the first place. The next milestone would be to achieve an improvement of the progression of the disease by medications offering the patients a higher degree of autonomy. Our long term goal, however, is the development of efficient therapeutic ways to cure BPAN.
The “positive” aspect about the BPAN progression is that the neurodegenerative deterioration is usually supposed to start only in early adulthood. Children of Milly’s age might therefore profit from a medication or a therapy. Adult patients would profit, too, as their symptoms would not get any worse. Ultimately, the research on BPAN could have a positive effect on the research of other recurrent neurodegenerative diseases like Alzheimer’s or Parkinson’s disease.
That is why we ask you to support Milly’s Mission against this cruel disease with your donation!
We decided against a professional fundraising website thus making it possible to use every single donated cent to combat BPAN.
Any small amount is important!