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.

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 ...

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 ...

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.

Genetic analysis explains rare disease severity, points to possible treatment by Washington University in St. Louis Normal lung tissue is shown on the left.

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 ...

Prince Frederik of Luxembourg died from a genetic disease called PolG, but experts hope his passing could bring more awareness to the rare condition that impacted his life. Prince Frederik ...

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