PLEASE FOLLOW THIS PAGE AND SHARE THIS TO AS MANY PEOPLE AS POSSIBLE in the hope of convincing Tom and the team at Allievex to give Sebastien access urgently. This is an ethical call to correct a wrong:
My son Sebastien has just turned 9 years old. He is reaching the end of the plateau period typical to this condition, a point when much more rapid degeneration takes its course with further loss of
his faculties, and a point of no return...equal to slow neurological regression and a short but miserable life. He has already responded positively to enzyme replacement therapy and should have been included on the present trial. However, in effect, because of a previous failed trial and the criteria of the present one, Sebastien’s life has been trashed. It is wrong to continue to refuse this young boy access to a product on which there is sufficient safety, efficacy and drug, together with a chance for a better quality of life, however long that may be. The pharmaceutical companies BioMarin and now Allievex have continuously changed the goalposts for four and a half years on the basis of wanting to wait to help a 'bigger group' and 'not having enough drug', whilst repeatedly extending the end date of the trial. This is not about prioritizing, this is not about one or the other, "this is about taking a look at yourselves in the mirror and knowing that you did everything you could do to offer Sebastien a greater chance for a better life", however long that may be. The more time goes by, the less likely the drug will make a difference. Sebastien's physician at Great Ormond Street Hospital (London) is also the Prime Investigator overseeing the study and is 100 percent in favor of proceeding. Everything is ready to go. We just need the good will and go ahead of the CEO of Allievex, the pharmaceutical company in question. The MHRA (UK regulatory equivalent of the FDA), as well as the European Agency of Rare Diseases (Eurordis) are all behind us. Please share this to as many people as possible in the hope of convincing Tom and the team at Allievex to give Sebastien access urgently.
