source: pixabay.com
Bioscience Companies in Ohio Take Aim at Rare Diseases
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…
The Major Ethical Dilemma Behind Rare Disease Drug Costs and a Bioethicist’s Solution
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…
<a href="https://pixabay.com/users/HeatherPaque/">HeatherPaque</a> / Pixabay
Family Affected by Batten Disease Raises Hundreds of Thousands Towards Research
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…
<a href="https://pixabay.com/users/typographyimages/">typographyimages</a> / Pixabay
Potential Gene Therapy for Late Infantile Batten Disease Earns Rare Pediatric Disease Designation
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…
Phase 1/2 Gene Therapy Trial for Batten Disease Now Recruiting
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…
<a href="https://pixabay.com/users/HeatherPaque/">HeatherPaque</a> / Pixabay
Teenager Raises Money for Sister With Batten Disease
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…
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…
<a href="https://pixabay.com/users/Pexels/">Pexels</a> / Pixabay
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…
Source: Pixabay
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…
Source: Pixabay
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…
Source: Pixabay
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…
source: pixabay.com
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…
Source: Pixabay
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…
Source: Pixabay
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…
Source: Pixabay
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,…
Source: Pixabay
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…
source: pixabay.com
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…
source: pixabay.com
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…
Source: Pixabay
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,…
source: pixabay.com
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…
Source: www.pixabay.com
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…
- 1
- 2
