Advisory Board

ALAN – Maladies Rares Luxembourg is a non-profit association  founded in 1998 and declared of public utility in 2000.

It is ALAN’s mission to improve the quality of life of individuals who are affected by a rare disease.

Canadian PKU and Allied Disorders Inc. is a non-profit association of volunteers, first organized in 2008 based in Toronto, Ontario. The Association is dedicated to providing accurate news, information and support to families and professionals dealing with phenylketonuria (PKU) and similar, rare, inherited metabolic disorders with the mission of improving the lives of people with PKU and allied disorders and the lives of their families.

It is DADA2 Foundation aim to find innovative ways to work together, accelerate research into the Deficiency of Adenosine Deaminase 2, raise awareness and improve the lives of patients around the world.

EAMDA works to support and improve the quality of everyday life of people with neuromuscular disorders (NMDs).

As a European umbrella organization, one of their main ambitions is to show everyone that regardless severe and progressive neuromuscular disorders, we can still contribute and benefit the society we live in.

Global Genes is a non-profit organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally, from early support and awareness through research readiness, using a collaborative approach that involves biopharma, researchers and funders, with data as a central core.

IPOPI is the Association of national PID (primary immunodeficiency) patient organisations.

IPOPI is dedicated to improving awareness, access to early diagnosis and optimal treatments for primary immunodeficiency patients worldwide through global collaboration with patients, doctors, politicians, regulators, pharmaceutical industry and other relevant stakeholders

The Myrovlytis Trust is a charity founded in 2007  to transform the outlook for rare conditions. With an initial focus on Birt-Hogg-Dubé Syndrome and osteosarcoma, the trust strategically funds research directed towards new treatments. The aim, to ensure that patients gain access to the same state-of-the-art technologies, breakthroughs and therapies as those with more common disease.

The Neuro Move CMT Association was founded in June 2019 in Romania and aims to inform, support and increase the quality of life of people affected by Charcot-Marie-Tooth Peripheral Neuropathy (CMT), other similar neuromuscular diseases and other types of disabilities, which hinder mobility and optimal psycho-social functioning of these people.

PROS Foundation is a non-profit organization passionate about helping rare diseases patients with their diagnostic journey, obtaining access to treatment and developing a path to clinical trials for new treatments. Although PROS Foundation is US-based, it also supports patients and groups in developing countries. 

Rett Syndrome Europe is the umbrella organisation for European Rett Syndrome patient organisations. Parent led, as volunteers we aim to raise awareness of the syndrome,  improve communication within the community and promote the interests of people with Rett syndrome and their families.

Sisters’ Hope Foundation is a non-profit organization dedicated to leukoencephalopathy with axonal spheroids and pigmented glia (ALSP)patients around the world. It is their goal to increase awareness and understanding of adult-onset ALSP, support those living with or caring for those living with ALSP and create connections for the ALSP community. Their mission, to see the first survivor of ALSP.

Team TRIO is a support network for families with loved affected by TRIO gene mutation as well as for professionals. The network offers impacted families support, resources, and advocacy, and wants to increase awareness in the medical arena and community at large.