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4-Year-Old from Florida Diagnosed with Extremely Rare Batten Disease
All parents have fears for their children, but few of them expect their child to be diagnosed with a disease so rare there are only 2-4 diagnoses for every 100,000…
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Rare Diseases: A Catch 22 Dilemma
A guest speaker at the 2019 Rare Disease Forum recently held at the North Carolina Biotechnology Center acknowledged that screening is necessary to justify the need for a particular…
<a href="https://pixabay.com/users/DarkoStojanovic/">DarkoStojanovic</a> / Pixabay
A Girl Diagnosed with Batten Disease got Treated with a Drug Developed Specifically for Her
According to a story from Technology Review, the story of Mila Makovec shows the potential impact that personalized medicine could have on patients while also revealing some of its limitations.…
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Personalized Medicine: A Drug Tailored for a Six Year Old With Batten Disease May Serve as a Template for Similar Rare Disorders
Mila Makovec began her life as a normal, healthy infant, and according to a recent article in Science Magazine, she continued along this path until reaching the age of…
<a href="https://pixabay.com/users/Free-Photos/">Free-Photos</a> / Pixabay
Boy From the UK With Batten Disease to Get Access to Critical Treatment
According to a story from derbytelegraph.co.uk, four year old Michal Myslicki, who was born with Batten disease, will soon get access to a critical treatment that will prolong his life.…
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NHS Finally Reverses Almost 2 Year Policy Against Offering Cerliponase Alfa to Batten Disease Patients
According to a recent publication from Express Digest, England's National Health Service (NHS) has reversed its earlier position on a rare disease drug after lengthy public debate, now agreeing to…
<a href="https://pixabay.com/users/Pexels/">Pexels</a> / Pixabay
Amicus Therapeutics Continue to Develop Investigative Treatments for Fabry, Pompe, and Batten Diseases
According to a publication from Fabry Disease News, Amicus Therapeutics has as many as 14 investigational drugs currently in development for the treatment of rare diseases including Fabry, Pompe, and…
<a href="https://pixabay.com/users/geralt/">geralt</a> / Pixabay
A Gene Therapy for the CLN6 Variant of Batten Disease Looks Promising
According to a story from globenewswire.com, gene therapy, a current hot topic in the medical field, is continuing to make headlines. The drug company Amicus Therapeutics recently announced that a…
Tickets are on Sale Now for Rare Disease Film Festival in Boston
Are we ready for our close-up? The second annual Selections from DISORDER: The Rare Disease Film Festival at Biotech Week Boston will be held at the Takeda Auditorium in Cambridge,…
<a href="https://pixabay.com/users/JESHOOTS-com/">JESHOOTS-com</a> / Pixabay
This Mother Isn’t Afraid to Move Abroad to Keep Her Daughter With Batten Disease Alive
According to a story from the St. Helens Star, Claire Riley, the mother of Kaycee Bradshaw, said in a recent statement that she would not hesitate to move out of…
<a href="https://pixabay.com/users/rawpixel/">rawpixel</a> / Pixabay
Drug Company and Foundation Partner Up to Develop New Treatment for Batten Disease
According to a story from Market Screener, the biopharmaceutical company Theranexus and the Beyond Batten Disease Foundation (BBDF) have recently signed an agreement to work towards the continued development of…
<a href="https://pixabay.com/users/geralt/">geralt</a> / Pixabay
Gene Replacement Research and the Hope to Cure Rare Disease
Researchers are studying gene replacement therapy in rare diseases at UT Southwestern Medical Center. Many rare diseases are caused from a defect in a single gene, so replacing the defective…
<a href="https://pixabay.com/users/Pexels/">Pexels</a> / Pixabay
Potential Treatment for Infantile Batten Disease Earns Fast Track Designation
According to a story from globenewswire.com, the gene and cell therapy company Abeona Therapeutics, Inc. has recently announced that its experimental gene therapy ABO-202 has earned Fast Track designation from…
<a href="https://pixabay.com/users/jameswheeler/">jameswheeler</a> / Pixabay
Family Calling for British Columbia to Approve Critical Batten Disease Treatment
According to a story from Global News, Charleigh, the three year old daughter of Trevor Pollack and Jori Fales, was recently diagnosed with CLN2 Batten disease, which is a rare…
<a href="https://pixabay.com/users/adamtepl/">adamtepl</a> / Pixabay
Family Seeks Handicap Accessible Home as Their Children Face Batten Disease
According to a story from CBS Minnesota, the Wahlstrom family has been hit hard by a deadly rare illness called Batten disease. Parents Todd and Laura's two children, Drew and…
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Parents of Kids Affected by Batten Disease Take Legal Action to Maintain Access to Critical Treatment
According to a story from Express Digest, Lucy and Mike Carroll don't know for how much longer their children will be able to live happy lives. This is because their…
<a href="https://pixabay.com/users/qimono/">qimono</a> / Pixabay
FDA Clears Company to Begin Trials for Experimental CLN1 Disease Gene Therapy
According to a story from apnews.com, the gene and cell therapy company Abeona Therapeutics, Inc., has recently announced that the US Food and Drug Administration (FDA) has given the company's…
Diagnostic Timeline for Lysosomal Storage Disorders like Fabry Disease Significantly Reduced in South Africa
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,…
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Sisters With Batten Disease Face Tragic Future After Rejection of Critical Drug
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…
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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…
