Public announcement of the foundation of the Global Association for Phenylketonuria (GAP) on July, 6th, 2018 at the occasion of the NPKUA-Conference in Atlanta, Georgia (USA)Tobias S. Hagedorn, Founding Trustee of GAP Introduction Good morning everybody! I am Tobias Hagedorn. Since 2001, I am the Secretary of the European Society for Phenylketonuria and Allied Disorders. Since
(Atlanta, July 7 2018) Today at the NPKUA Conference in Atlanta, Georgia, the Global Association for Phenylketonuria (GAP) was unveiled. Representatives from patient groups across four continents have been working towards this dream since it was first conceived of in Dublin, Ireland 2016. Representative from Germany, Tobias Hagedorn stated, “I am proud and deeply humbled that it falls to
MDDA welcomes the decision by the Pharmaceutical Benefits Advisory Committee (PBAC) to defer the listing of Kuvan (sapropterin hydrochloride) on the Pharmaceutical Benefits Scheme (PBS) for Australians living with phenylketonuria (PKU). Whilst it is disappointing not to have received a positive recommendation, the deferral means the PBAC recognises the benefit of Kuvan for some PKU
MDDA President, Monique Cooper presented at Parliament House, Canberra, on the 15th February 2018 with Nicole Millis from Rare Voices Australia. Here is Monique’s speech. Thank you Nicole….Whilst I am here today representing the MDDA as their president, I also stand before you as a parent of a child diagnosed with a rare disease known
Families living with rare diseases are being invited to complete an online questionnaire about preconception carrier-screening (please see web link below). The questionnaire is part of the Australian Genomic Health Alliance (AGHA) preconception carrier screening flagship program being drafted currently. The study is being conducted by a multidisciplinary research team led by Professor Nigel Laing
A BIG thank you to all of our Pledgers and Players who took part in the 2018 Great Protein Challenge held in February. Your support means so much to us and all those people living with PKU who have to ‘go low pro’ every single day. We raised over $16000 in our first year launch
Media Release – When healthy, nutritious foods make you sick … the dilemma facing 1,600 Australians
The Great Protein Challenge to put Aussies in the shoes of those living with a debilitating rare disease From 1 February 2018, Australians will be asked to give up protein for 24 hours to help raise much needed awareness and funds to support those living with a debilitating, rare genetic metabolic disorder – called Phenylketonuria or
Kuvan (sapropterin dihydrochloride) has been submitted to the Pharmaceutical Benefits Advisory Committee (PBAC) for consideration to be subsidised on the Pharmaceutical Benefits Scheme (PBS) for responsive patients with PKU. KUVAN is the first and only prescription medicine available for PKU. MDDA is extremely supportive of this announcement as we have made several approaches to the
Our friends at The Grand Hotel Warrandyte have released their latest specialised menu for IEM families and those requiring a low protein diet. The new menu offers a selection of delicious, restaurant quality low-protein menu options including flavoursome starters, mains & desserts. Make sure if you are in Victoria you pay The Grand a visit
LCW spoke to Bianca Albanese, Biomedical Engineer and PKU Advocate about what it means to be a woman in engineering and living with PKU.
The MDDA is very excited and proud to announce our participation in the first global association for PKU. In collaboration with leading International PKU associations and groups, the MDDA is playing a very active role in the establishment of this new global association and further information on our strategic vision can be found herein.
MEDIA RELEASE – Australian first – restaurant creates unique dining experience for families with rare disease
MEDIA RELEASE – FOR IMMEDIATE RELEASE Australian first – restaurant creates unique dining experience for families with rare disease The Grand Hotel Warrandyte today launched a specialised menu for families with rare Inborn Errors of Metabolism (IEM), such as PKU (phenylketonuria), to mark PKU Awareness Month. Eating out for those with an IEM is often