Results 171 to 180 of about 1,318,634 (291)
ABSTRACT Objective Spinocerebellar ataxias (SCAs) are a group of genetically heterogeneous neurodegenerative diseases causing progressive deterioration and reduced quality of life. Therapeutic advances have been limited by a lack of sensitive anatomic, functional, or diffusion imaging‐based biomarkers.
David J. Arpin+11 more
wiley +1 more source
Research and development investments for biologics independently developed by US biotechnology startups, 2017-2023. [PDF]
Odouard IC+4 more
europepmc +1 more source
ABSTRACT Objective Malignant gliomas pose significant therapeutic challenges. This study aimed to identify and characterize a novel chimeric RNA in glioma and assess its clinical and functional significance for precision treatment. Methods The C19orf47‐AKT2 chimeric RNAs were identified through RNA sequencing and validated by polymerase chain reaction.
Zihan Wang+11 more
wiley +1 more source
Real‐World Comparison of High‐Efficacy Versus Non‐High‐Efficacy Therapies in Multiple Sclerosis
ABSTRACT Objective The choice of the first disease modifying treatment (DMT) in multiple sclerosis (MS) is a topic of great interest, and whether high‐efficacy DMTs should be the first choice remains debated. We compared treatment outcomes (no evidence of disease activity [NEDA] and its components) between treatment‐naïve relapsing–remitting MS (RRMS ...
Sarmad Al‐Araji+9 more
wiley +1 more source
Initiating the commercialization of genetically modified staple crops in China: domestic biotechnological advancements, regulatory milestones, and governance frameworks. [PDF]
Mou T, Song Q, Liu Y, Song J.
europepmc +1 more source
Repeat Expansions in PLIN4 Cause Autosomal Dominant Vacuolar Myopathy With Sarcolemmal Features
ABSTRACT Objective We aim to describe and characterize two unrelated Spanish families suffering from an autosomal dominant autophagic vacuolar myopathy caused by repeat expansions in PLIN4. Methods We evaluated the clinical phenotype and muscle imaging, and performed a genetic workup that included exome sequencing, muscle RNAseq, and long‐read genome ...
Laura Llansó+17 more
wiley +1 more source