Spark Therapeutics launches gene therapy clinical trial for late-onset Pompe...
Pompe disease is a hereditary genetic disorder caused by a deficiency of acid alpha-glucosidase leading to build-up of glycogen in the lyosomes, which then causes cell damage in various tissues, in...
View ArticleA new 3D model for myotonic dystrophy
Myotonic dystrophy is a hereditary degenerative neuromuscular disease that occurs mainly in adults, affecting about 50,000 people only in Spain.
View ArticleStudy reveals the role of SPOP protein in an inherited form of ALS
The damaging effects of toxic proteins created in one inherited form of amyotrophic lateral sclerosis (ALS) are mediated by a protein called SPOP, according to a study published in the Proceedings of...
View ArticleIdentifying an important protein for motor coordination
News-Medical speaks to Professor Jorge Ruas about how he has identified an important protein involved in motor coordination.
View ArticleNew consensus-based recommendations designed to improve quality of life for...
Many more adults with Duchenne muscular dystrophy (DMD) are living longer thanks to improvements in treatment, however international standards of care have not yet fully addressed the complex needs of...
View ArticleUT Southwestern researchers reveal the physical mechanism behind a...
Using UT Southwestern's Cryo-Electron Microscopy Facility, researchers for the first time have captured images of an autoantibody bound to a nerve cell surface receptor, revealing the physical...
View ArticleVaccines and monoclonal antibodies being developed for respiratory syncytial...
A new study reviews recent advancements made in the development of RSV immunoprophylactic measures.
View ArticleUsing human iPSC-derived cells to revolutionize the field of...
In this interview, we speak to Axol Bioscience about their human iPSC-derived cells and how they have the potential to revolutionize the field of neurodegenerative disease.
View ArticleFirst successful Phase 2 trial uses cutting-edge RNA CAR-T therapy to treat...
Cartesian Therapeutics, a clinical-stage biotechnology company pioneering cell therapies for autoimmune diseases, announced today the publication of a landmark paper in The Lancet Neurology.
View ArticlePatient-specific muscle models pave way for personalized neuromuscular therapies
Scientists have so far identified around 800 different neuromuscular diseases. These conditions are caused by problems in the way muscle cells, motor neurons and peripheral cells interact.
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