Gene Therapy Progress for Batten Disease and Sanfilippo Syndrome
REGENXBIO is a biotechnology company that has been developing a technology platform for gene therapy called NAV. AAV9 is one of the vectors being tested with NAV technology as a…
New Batten Disease Treatment Just Received Orphan Drug Designation!
Batten disease is a lysosomal storage disorder. It's neurodegenerative, meaning it results in the deterioration of neurons. One of the most common forms of this disease is caused by mutations in the…
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Mila’s Experimental Treatment for Batten Disease
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…
Scientists Discover a Better Batten Disease Model In Primates
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…
Competing for a Cure: How a New Fitness Event Raised Money for Batten Disease Research
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…
A 4-Year-Old Was Learning to Speak, But he Forgot his First Words Thanks to Batten Disease
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…
Batten Disease Takes Two Children in the Same Family
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…
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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…
Study Finds That an Enzyme Therapy for Batten Disease is Effective
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 Being Developed to Treat Batten Disease Has Been Granted Orphan Drug Status in Europe
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…
Grief, Obstacles, and Hope: A Mother’s Will to Create Choices
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…
Photo courtesy of Gail Rich
These Sisters With Batten Disease Are Finally Getting Treated at the Same Hospital
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,…
HOPE on the Green: A Hole-In-One for Batten Disease Research
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…
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A Treatment for Batten Disease Was Given Rare Pediatric Disease Designation from the FDA
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…
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Family Spreads Awareness After Their Son Was Diagnosed With Batten Disease
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…
An Experimental Drug for Batten Disease Just Got Orphan Drug Designation From The EMA
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…
Photo courtesy of Julia Vitarello
A Girl From Colorado is Receiving An Experimental Treatment for Her Batten Disease
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,…
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The FDA Commissioner Says He Plans to Combat High Drug Prices
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…
Family Gets a New Home to Help Care for Their Two Children With Batten Disease
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…
A Boy With Batten Disease Touches America With Viral Video Listening To An Ed Sheeran Song
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…
What Does it Mean When We’ve Identified Batten Disease Biomarkers?
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…
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This Voice is Standing Up for the Rarest of Patients in Washington
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…
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A Life That Didn’t Go According to Plan: A Batten Disease (BD) Story
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…
Batten Disease In the News
Want some more news, events and announcements on Batten disease? We got 'em! Don't forget to share!
Editor’s Choice: IDF Sessions on CGD, IPF Progress and FDA Approval
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…
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