HOW YOUR DONATION WILL HELP
We are a group of moms and patients fighting for our children. We have an expert team helping us be the voice for the patients to save access to these two experimental medicines - called adrabetadex and arimoclomol.
All funds go directly to Hope For Marian Foundation, a charitable 501(c)(3), and will be used so our team of regulatory, drug development, legal, communication and rare disease experts team can champion our children's need to safeguard access to these medicines through advocacy and education. Hope For Marian Foundation has the guidance of a NPC parent advisory board. Ultimate decision making authority and all control of funds will rest with Hope For Marian in allocating the funds to support access to these medicines.
We need OUR stories to be heard that these medicines work. These are our children. Please help us save them.
Sign Our Petition
Read Our Communication With FDA
Watch Our Video and Media Supporters