The Problem Lysosomal storage disorders (LSDs) affect approximately one out of every 7,000 people. There are around 50 different types of LSDs. These include Fabry disease, Batten disease, Gaucher disease,…
According to a story from Mirror Online, parents Gail and Matthew Rich were devastated when they heard the news that a critical treatment for Batten disease was rejected for coverage…
According to a story from BioPortfolio, Rare Disease Day, which took place on February 28th, 2019, serves as a time of reflection on the progress that has been made in…
Avraham Steinberg recently spoke at RARE2019, the 2nd International Congress on Advanced Treatments in Rare Diseases. It was held on March 4th and 5th in Vienna, Austria. His talk focused…
According to a story from Statesman Journal, nine year old Haley Pollman isn't so different from most other girls her age. She loves dresses, Barbie dolls, and animals, and still…
According to a story from BioSpace, the biotechnology company REGENXBIO, Inc. recently announced that its experimental gene therapy candidate called RGX-181 has been granted Rare Pediatric Disease designation from the…
Batten Disease There are 13 different forms of Batten Disease defined by the medical community. Each presents similarly but there are differences in the severity of symptoms as well as the…
According to a story from CBS News, Garland Benson, age 14, has undertaken a daunting task in order to save the life of his older sister Christiane. She was born…
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…
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…
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,…
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