YourTango19h
The Symptom I Ignored At Cheerleading Practice Turned Out To Be A Rare, Life-Altering Genetic Disease
FA is a progressive neuromuscular disease that causes loss of coordination and mobility. My parents got my test results the ...
Science Daily2d
Gene editing approach paves the way to first-in-human clinical trial for rare genetic disease
New research demonstrates the potential of precise genome editing technologies, called adenine base editors, to correct disease-causing mutations in stem cells from patients with X-linked chronic ...
Medical Xpress on MSN1d
Little-studied RNA might be key to regulating genetic disorders like epilepsy and autism
"The unique mechanism we've identified here suggests that there are more long non-coding RNAs underlying rare genetic ...
Business Wire2d
Savara Announces the Rare Lung Disease, Autoimmune Pulmonary Alveolar Proteinosis (aPAP), is Highlighted on a New Episode of “The Balancing Act ® ” Airin…
Savara Inc. (Nasdaq: SVRA) (the Company), a clinical stage biopharmaceutical company focused on rare respiratory diseases, ...
Neighbours rally around 10-month-old boy with rare — and terminal — genetic disease
Residents who live in Country Lane Estates trailer park south of Calgary helping raise money for family of a 10 month old boy ...
The Peninsula3d
Sidra Medicine pioneers gene therapy in Qatar for children with rare genetic disorders
Sidra Medicine s Genetic and Genomic Medicine clinic is providing life altering gene therapy for rare genetic disorders. In a ...
Rare disease ruined his life
A TEEN has spent the last 10 years in a wheelchair after he was diagnosed with a rare genetic muscular disease known as ...
HealthDay on MSN14h
Newborn Genome Analysis Spots More Health Issues Than Standard Screening
DNA analysis of newborns can detect many more preventable or treatable health problems than standard newborn screeni ...
STAT3d
In a bid to treat their children’s rare diseases, these three parents founded companies
At #STATSummit, three parents discussed the challenges they've faced trying to find treatments for their children with rare ...
Global News6d
Neighbours rally around 10-month-old boy with rare — and terminal — genetic disease
That’s because Tucker suffers from a rare and terminal disease called D-bifunctional protein deficiency which, according to the U.S. National Library of Medicine, is a disorder that causes ...
Researchers identify possible treatment for rare disorder CTNNB1 syndrome
Researchers at Tufts University School of Medicine and the Graduate School of Biomedical Sciences (GSBS) have identified a ...