**UPDATE - 02/07/2019**
To the disappointment, disbelief and frustration of Max's family and network of friends and supporters, the devastating news that the NHS declined Max's individual funding request (to obtain the critical enzyme replacement therapy) was received earlier today. It's expected the NHS will provide a written statement in the next seven days. Just to point out, 11 other Batten disease sufferers are currently receiving the treatment under the drug trial or on compassionate grounds. A gene therapy trial is starting late next year and if it proves successful, it promises to cure the disease, which would be a dream come true for patients like Max.
The news has still not fully settled. It's hard to put into words the effect this has had on the family yet again. It is crucial for Max to get access to the treatment at the first opportunity - to enable him to be spared the worst effects of the debilitating disease and to buy precious extra time for him, his family and friends to experience together. Sadly, time is not on his side and the financial requirements to achieve this are far outside of the family's ability to pay. Routes to legally appeal the decision and also to privately fund the treatment are being sought by the family as well as medical professionals. Opportunities for Max and his father Simon to move abroad for treatment are being investigated.
In the meantime, the family and the wonderful, growing support network of friends and supporters have joined forces and continue in our efforts to raise awareness of Max's case and fundraise to support his care and treatment and help fund whatever decision will have to be taken to save Max.
If you are in a position to help in whatever way or spread the word to those who are, please, please do contact us. For those, who are able to help financially, however large or small, please do donate to the this crowdfunding site. Any help at all is immensely appreciated by the whole family in these impossibly devastating circumstances. Huge thank you to you all in advance X
#saveourmax
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In my previous project I introduced you to my darling nephew Max (aged 8) who was diagnosed with the CLN2 type of fatal Batten disease earlier this year. Cick here for the previous fundraising page set up for darling Max and his story.
With your generous support we managed to hep fund a specialized mobility trike, sports equipment and aids, travel expenses for medical consultations and obtain legal advice. House modifications to make disabled access are much needed and on the list of things to do in the next couple of months.
The family continue to fight for national coverage and desperately hope they can get access to enzyme therapy treatment Max urgenty needs through the NHS. Some progress has been made, although for Max who continues to deteriorate rapidly day by day the treatment cannot come soon enough.
The enzyme treatment will sadly not cure Max but will help save as much of the happy boy we know and love and will stabilise his condition for the time he has remaining. For those reasons the family are looking to make the heart wrenching decision for Max and his dad Simon to leave his mum and younger brother Felix to move to Holland where there is a chance that they can secure funding for his regular fortnightly treatment.
The American drug company even offered to provide a free trial for Max (who is one of the two patients currently not receiving the drug) but NHS England has disappointingly rejected the offer and is not prepared to engage in further negotiations. You can read about it here. Max's local MP Jacob Rees-Mogg has been trying his best to intervene and has been pleading with the House of Commons to take action in his favour. His moving speech can be viewed here. Here is also a link to a recent article in the national press covering the impossible challenge my sister and her family face.
If you are in a position to help in whatever way or spread the word to those who are, please do contact us. For those, who are able to help financially, however large or small, please do donate to this crowdfunding site. Any help at all is immensely appreciated by the whole family in these impossibly devastating circumstances. Huge thank you to you all in advance.
#saveourmax
Appeal to the UK Government to save the lives of children with CLN2 Batten Disease - May 2019.