Last time Patient Worthy wrote about Mila Makovec, she was seven and beginning a cutting-edge experimental treatment for her Batten disease. It’s now nine months later, and Mila is showing…
According to a report by Science Daily, researchers recently discovered a disease in monkeys which imitates human Batten disease. Establishing the closest analog to the condition in humans, the version…
A new fitness competition held its first contest this April in Sunderland. A contest based around individual functional fitness, First Means Everything donated a percent of its sign-up fees to…
According to a story from the Leamington Observer, four-year-old George Young's first words were 'mama' and 'papa,' but he did not know how to say them for very long. George's…
No one should outlive their child. Losing two children to the same rare disease is a pain that most parents can never understand and hopefully never will. According to a…
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…
A study has just been published in the New England Journal of Medicine (NEJM) that found that the drug Brineura slows down the deterioration of language and motor function in…
A gene therapy called ABO-202 that is being developed to treat Batten disease has just been granted Orphan Drug Designation by the European Medicines Agency (EMA), reports BioPortfolio. This follows…
For so many in the rare disease community, courage isn't a just a virtue - it's a necessity. Many have to overcome odds and obstacles that most others don't have…
Sisters Nicole and Jessica Rich, both diagnosed with Batten disease, will finally be able to receive treatment at Great Ormond Street Hospital in London. Nicole, who is six years old,…
April 9th is promising to be a beautiful day this year. By then, spring should be in full swing. For some people that phrase rings quite literally. With the return…
According to a story from pharmpro.com, the pharmaceutical company Abeona Therapeutics announced that it has been given Rare Pediatric Disease Designation for its ABO-202 program by the U.S. Food and…
According to a story from 13abc.com, a family is working to spread awareness about Batten disease after second grader, Charles Klein was diagnosed with the condition. The disease has no…
According to an article from kait8.com, the pharmaceutical company Polaryx announced that its experimental drug PLX-200 has received Orphan Drug Designation from the European Medicines Agency (EMA) for the treatment…
According to an article from Times-Call Regional News, a seven year old girl named Mila Makovec has just begun an experimental treatment for Batten disease. Originally from Boulder County, Colorado,…
According to a story from CNNMoney, Scott Gottlieb, the current commissioner of the US Food and Drug Administration (FDA), said that he plans to curtail high drug prices and go…
The Wahlstrom family of four, is getting a miraculous gift to help care for their two children suffering from Batten disease, reported CBS Minnesota. With the love of their local…
Music is sometimes more powerful than we even realize on the daily. Music keeps little 6-year-old Ollie Carroll going, even though he understands his life will be short. Ollie and…
Batten disease, also known as neuronal ceroid lipofuscinoses (NCLs) is a fatal, inherited disorder of the nervous system. It involves a build up of lipopigments that accumulate and kill brain…
Batten disease is one of the rarest diseases in the world. In fact, it only affects about two to four out of every 100,000 births in the United States, which…
In June 2017, a film was released showcasing the story of a young girl named Kennedy Hansen who had Batten disease—a disease that causes a damaging buildup of lipofuscins in…
Want some more news, events and announcements on Batten disease? We got 'em! [one_half] [/one_half] [one_half_last] Pioneering Brain Surgery May Give This Toddler More Time FDA Press Release…
Happy Friday Patient Worthians! You might notice that the headline has a lot of acronyms in it... ah, welcome to the rare disease world! We DO have answers for what all of that…
The drug, Ceriliponase alfa from BioMarin, helped in a condition that has led inexorably to disability and death: Batten Disease – specifically CLN2. Families of kids facing this prognosis are…
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