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Rare Ireland

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Alan & Michelle embark on a journey to advocate for those affected by multiple sulfatase deficiency

Alan Finglas, alongside his wife Michelle, embarked on a journey to advocate for those affected by multiple sulfatase deficiency (MSD) after their son Dylan’s diagnosis in 2015. At that time, there was no dedicated charity pursuing research on MSD anywhere in the world. Alan is known for his sheer determination, practical mindset, fundraising abilities, strategic planning, his ability to help secure grants, and being able to discover and foster collaborations. The MSD Action Foundation & SavingDylan.com have been pivotal in reshaping the landscape of MSD research.

Alan Finglas remarked, ”Receiving this award is one of the greatest honours of my life. Part of it belongs to the MSD researchers, the MSD patient families around the world that helped to make an impact on MSD so far and those that donated to the cause”.

As part of his work with REMEDi4ALL, Alan was instrumental in establishing a cooperative effort with AbbVie to further clinical research on tazarotene as a potential treatment option for MSD. This project involving Fraunhofer ITMP, University Medical Center Göttingen and EATRIS will benefit greatly from access granted by AbbVie to critical efficacy, safety and clinical development data on a formulation of tazarotene suitable for treating MSD patients.

Dr. Lars Schlotawa, Paediatrician/Consultant for Paediatrics at the University Medical Centre Göttingen and REMEDi4ALL Partner, praised Alan’s dedication, stating, “Alan’s tireless advocacy has propelled MSD research forward, offering hope to countless families worldwideMSD is no longer the ultra-rare, forgotten disease because Alan put MSD onto the big global stage. ”

The 2024 Patient Advocate Leader Award was presented at 7:30 AM PST on Tuesday 6 February 2024, at the 20th annual WORLDSymposium in San Diego, California. A video tribute has been released, capturing Alan’s unwavering spirit and the impact of his advocacy efforts. released, capturing Alan’s unwavering spirit and the impact of his advocacy efforts. You can watch the video here including his acceptance speech at the end:

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About MSD Action Foundation / SavingDylan.com
MSD Action Foundation’s & SavingDylan.com are one in the same body whose main goal is: ‘To promote and support research advancements that will lead to positive clinical outcomes, and quality of life, for patients suffering from Multiple Sulfatase Deficiency’. They were the first ever dedicated patient organisation advocating for MSD when they were established in 2015. The goals of the foundation are to support translational research in order to further breakthroughs in MSD research, to create more public awareness of MSD, and to act as a voluntary support and information exchange network for those affected by MSD and their families. Find out more here: www.savingdylan.com/about-the-foundation

About REMEDi4ALL
REMEDi4ALL is an EU-funded research initiative that is driving forward the repurposing of medicines in Europe. The project is expected to make a major leap forward in drug repurposing, or finding new therapeutic options for existing drugs, in areas where there are high unmet medical needs. It is a 5-year, €25M Horizon Europe project that aims to develop an innovation platform supporting promising, high impact drug repurposing projects championed by patients in any phase of development and disease area. A global community will be established that contributes to informing and shaping policy and advancing debate and knowledge exchange worldwide. The multidisciplinary consortium involves 24 European organisations, led by EATRIS, with the common goal of making cost-effective repurposed medicines more widely available. University Medical Centre Göttingen as one of the world leading centres for MSD research and patient care is a partner of REMEDi4ALL. Find out more here: www.remedi4all.eu

Rare Disease Strategy

A new Rare Disease Strategy is being undertaken by a steering committee consisting of members from the HSE, Dept of Health, IPPOSI, RDI and two patient advocates.  This new strategy will replace the previous Rare Disease Strategy (2014 -18).

The Rare Disease Patient Forum is being established to ensure the voices and lived experience of people living with rare diseases and their families remain central to the development of this new Rare Disease Strategy. The Patient Forum will be chaired by Vicky McGrath, CEO Rare Diseases Ireland and Dr Derick Mitchell, CEO IPPOSI. The input collected through the Patient Forum will feed directly into the work of the Steering Group in developing the new Strategy. The first meetings took place in February.