Delayed Decision on Batten Disease Drug Leaves Girl’s Life in Jeopardy

According to a story from blackpoolgazette.co.uk, the NHS has decided to postpone a decision to approve a drug for the treatment of Batten disease. This illness, which is fatal is often fatal by late teens or early twenties, or earlier if left untreated, has only a single treatment on the market that is designed to treat it called Brineura. The family of Saffa Shehzan, a five-year-old girl who has the disease, were hoping for the treatment to gain approval. She already has suffered severe decline because of her illness.
Batten disease is a disease that affects the nervous system. The illness is caused by a genetic mutation. There is come confusion as to how this term is used; some doctors use the term Batten disease to refer to a specific type of juvenile neuronal ceroid lipofuscinosis. However, it is also becoming more common to use the term to refer to this entire group of diseases. Symptoms include seizures, vision problems, changes to behavior, slow learning or regression, loss of coordination, and repetitive speech. Saffa was diagnosed with the CLN2 variant, which occurs in the first few years of life. Generally, functionality decreases rapidly over the span of a few years; many patients do not survive past age ten. To learn more about Batten disease, click here.

By now, her parents fear that even if the drug does get approved in the next few months, it may be too late to preserve most of Saffa’s functioning ability. The family needs the drug as soon as possible in order to slow down the progression of the illness. She is already unable to walk or eat, and her vision will be gone soon too. Now, her father Majid is desperately hoping that they can get Saffa into a trial in order to get access to the drug sooner. The decision to approve the drug has been delayed until September, which would probably be too late for Saffa.

The main reason that the drug has not made it onto the NHS so far is because of its staggering cost: at least £500,000 for a year of treatment. However, Brineura has managed to gain approval both in the U.S. and in the European Union. Hopefully, there will be an opportunity for Saffa to get treated earlier; it’s a race against time.

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