BioMarin's Narrow Moat Supported by Rare Disease Therapies and Developing Pipeline
BioMarin's gene therapy pipeline has had setbacks, including a three-year delay in US approval of Roctavian and stalled development of PKU gene therapy BMN 307 as the Food and Drug Administration ...
AZoLifeSciences on MSN2d
Using CRISPR-Cas9 Gene Editing for Chromosomal Rescue in Down Syndrome
Gene editing techniques may eventually allow trisomy to be treated at the cellular level, according to an in vitro ...
Labroots2d
Assessing Gene Therapy Approaches for Down Syndrome
Decades ago, researchers showed that Down syndrome (also known as trisomy 21), was caused when individuals carried an extra ...
Health Rounds: Gene editing may hold key to preventing Down syndrome
Gene editing techniques may one day allow for the removal of the extra chromosome that causes Down syndrome, early ...
News Medical3d
Gene editing shows promise for treating trisomy at cellular level
Gene editing techniques may eventually allow trisomy to be treated at the cellular level, according to an in vitro ...
Genetic study links defects in sugar digestion to irritable bowel syndrome
Sucrase-isomaltase (SI) is an intestinal enzyme critical for the digestion of dietary carbohydrates, particularly sucrose and ...
'The biggest thing about having a child with Down’s syndrome is you can’t be just a parent'
The couple from St Brides Major in the Vale of Glamorgan knew little about Down’s syndrome but decided there was no decision ...