Researchers at the Sanford Stem Cell Institute have taken the fight against rare diseases to the lab, to patients’ bedsides ...
QRL-201 shows potential to slow ALS progression by restoring a key protein needed for nerve health, according to data from a Phase 1/2 trial.
12h
First successes in the development of a gene therapy for incurable LAMA2-related muscular dystrophy
Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal muscle disease in children. The study shows in animal models that a single ...
Her son Ben, she learned, had mucopolysaccharidosis (MPS) type 2, or Hunter Syndrome, one of a group of rare diseases that is ...
Design Therapeutics (NASDAQ:DSGN) outlined progress across three clinical-stage programs and discussed upcoming catalysts during an Oppenheimer healthcare conference presentation led by CEO Pratik ...
A researcher at the University of Missouri has made a promising breakthrough in the quest to help people with amyotrophic lateral sclerosis (ALS), the neurodegenerative disorder commonly known as Lou ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results