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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…
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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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…
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.…
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…
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…
Families in England affected by Batten disease are struggling to access effective treatments in their home country. Nicole and Jessica Rich are two young girls who live with their families…
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…
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,…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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