As reported in Stem Cells News; finding the right donor for people in need of bone marrow transplants can be very tricky. This is because finding the correct match between…
The 2020 Virtual Family Conference June 25-27, 2020 This event features live streamed webinars featuring leukodystrophy researchers and relevant content for leukodystrophy patients and their families. The conference will also…
During the 10th European Conference on Rare Diseases & Orphan Products, hosted by Eurodis, many people advocated for improved newborn screening panels. In particular, these panels should include more testing…
As reported in PressReleasePoint, Researchers at the University of Sheffield have found a clue to the cause of a set of rare neurological disorders known as leukodystrophies- an issue with…
This was originally published as the Seventh Newsletter for the Leukodystrophy Resource Research Organisation (LRRO) on April 19th 2020. You can read more from their newsletter here. To state the blatantly…
According to a story from koco.com, the community of Ada, Oklahoma, recently held a birthday celebration for a local affected by Alexander disease. Hunter Goodwin recently turned 18 years old,…
Rare Disease Institute The Rare Disease Institute (RDI) is a new initiative aimed at improving collaboration between rare disease stakeholders. The aim is to facilitate faster diagnosis and better care…
When your child receives a rare diagnosis that needs instant care, you don't have time to contemplate finances and plan out your budget. You just have to act. That was…
By Caitlin Seida from In The Cloud Copy Although it’s already known that the cause of Alexander disease (AxD), a type of leukodystrophy, is a misfolded protein known as GFAP.…
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A recent article in the Taunton Gazette describes the dilemma faced by the parents of four-year-old Jaxtien Miller who is a metachromatic leukodystrophy patient waiting for a stem cell…
Finding the correct doctor for treatment can be difficult, especially if one has a rare disease. Because few doctors specialize in rare diseases, they are often spread across the…
The development of treatments and medicines for diseases can take a long time. These treatments come from many hours of research, studies, trials, and waiting for approval from the…
According to a story from news-medical.net, the National Institutes of Health (NIH) has doled a total of $31 million on grants this year to 20 different research teams. The goal?…
According to a story from CBS Philly, Jackson Guernsey, who is just two years old, was diagnosed with rare leukodystrophy just six months ago. While the disease has been taking…
Although each disease on its own may be rare, a huge number of people are living with a rare disease-- and half of these people are children. About 30 percent of these children…
Leukodystrophy is the term used for a group of rare, progressive brain disorders which cause the brain's myelin sheath (the nerves protective covering) to either develop improperly or be destroyed…
Cedar Rapids, Iowa is rallying to help a one-year-old girl who has been diagnosed with Caravan disease, a devastating disorder that often leads to premature death, within mid-childhood. What is Canavan…
This is Part 2 of Alexander's Story, click here to read Part 1. During Alex’s journey Jhoanny had gotten some information from NORD’s website and thought about getting involved. After…
Jhoanny’s son Alexander Matthew was born perfectly healthy in 2008. When Alexander was three, Jhoanny noticed he seemed to be drinking a lot of water and sweating a lot. Since…
Meet Ruby, a beautiful 3 year old who was diagnosed with hypomyelination with atrophy of basal ganglia and cerebellum, or H-ABC, shortly after her birth. H-ABC is a type of leukodystrophy…
The 2018 Scientific Symposium and Family Conference This conference will consist of a scientific symposium program that will allow medical professionals and researchers to present the latest research in the…
According to a story from abc.net.au, ever since Massimo Damiani was first diagnosed with a leukodystrophy, his parents, Sally and Stephen, have been committed to promoting research efforts and new…
Eva-Rose Sturgess is eighteen months old. She faces a disease for which there is no known cure: leukodystrophy. Her parents felt like their world came apart at the seams when…
A new treatment under development has produced promising results in patients with blood cancers and is now being tested in inherited metabolic disorders, reports Financial Buzz. Inherited metabolic disorders are…
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