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Catherine and the Rich family used the meeting with the Health Secretary to urge him to convene an emergency meeting with NICE, NHS England and BioMarin - the manufacturer of the treatment - to find a way of overcoming the barriers to making Brineura available on the NHS, and to look at whether an exception should be made in this case given the very small number of children affected by CLN2 Batten Disease who want to access the treatment on the health service.
"Funds donated will go into the Mia Johannah Trust, which will distribute in Mia's name all the monies collected to the charities chosen by Peta and Hamish such as: Riding for Disabled Association of Australia, Bear Cottage, GuideDogsAustralia and Batten Disease Support and Research Association."
In BEFORE November 2013 neurologists took blood tests to see if they could identify a faulty gene found in Batten Disease sufferers.
Batten disease is a rare, fatal genetic disorder that affects the central nervous system of children.
Jessica, three, and Nicole Rich, six, both have Batten Disease
Gail and Matthew have a team of around 20 people running for The Nicole Rich Foundation, which raises funds for the Batten Disease Family Association.
Ollie and Amelia - who have two older siblings - from Poynton in Cheshire, both have a rare genetic condition called late-infantile Batten disease. As they get older, they will lose their speech, mobility and eyesight, and are unlikely to live past age 12.
Biotechnology company BioMarin Pharmaceutical (Nasdaq:BMRN) reported on Wednesday that it has received US Food and Drug Administration (FDA) acceptance for the review of the submission of a Biologics License Application (BLA) for cerliponase alfa, an investigational therapy to treat children with CLN2 disease, a form of Batten disease.
Little Nathan Watson suffers from infantile Batten disease - a nerve condition which has left him so sick he has lost his sight, is unable to communicate and has constant seizures.
Amicus announced the initiation of a Phase 1/2 clinical study to evaluate the safety and efficacy of a single intrathecal administration of adeno-associated virus serotype 9 AAV9-CLN3 gene therapy in children with CLN3 Batten disease. Batten disease is the common name for a broad class of rare, fatal, inherited disorders of the nervous system also known as neuronal ceroid lipofuscinoses, or NCLs.
Gail Rich's daughters Nicole, five, and Jessica, two, both have Batten disease, an incurable condition which reduces life expectancy to an average of just 10 years old.