and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Japan, Europe, and internationally. Its biologic products include ...

A primary growth driver for the Mepsevii market is the rising prevalence of mucopolysaccharidosis (MPS) disorders. These genetic conditions ... facilitating access to rare disease treatments.

and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Japan, Europe, and internationally. Its biologic products include ...

Diagnosed with a rare condition In February 2024, when she was five, Liliana was diagnosed with Morquio syndrome — also known ...

Data from the first patients enrolled into Regenxbio's trial of its gene therapy for rare inherited disease mucopolysaccharidosis ... that could correct the genetic defect n MPS I, but that ...

In February 2024, when she was five, Liliana was diagnosed with Morquio syndrome — also known as mucopolysaccharidosis type IV — a rare genetic condition that prevents ... Lilianna is the first in NL ...

POLG-related diseases disrupt the function of the mitochondria, or "powerhouses" of the cell — starving them of energy.

Researchers at Washington University School of Medicine in St. Louis have uncovered why some patients with a rare genetic ...

A new study involving UCL researchers has found 69 previously unidentified genetic determinants of rare disease, including uncommon forms of epilepsy and schizophrenia. The research, published in ...

Cats are beloved family members in tens of millions of households, but we know surprisingly little about their genes.

Affected populations: POLG-related diseases are a group of rare, incurable conditions caused by inherited gene mutations. These disease-causing mutations are carried by up to 2% of people of ...