The treatment of this rare genetic disease is based on ... in patients are beta thalassemia and two metabolic diseases of childhood, mucopolysaccharidosis type 6 and type 1. In addition, within ...

The treatment of this rare genetic ... two metabolic diseases of childhood, mucopolysaccharidosis type 6 and type 1. In addition, within the Telethon institutes a targeted therapeutic strategy is ...

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.

Diagnosed with a rare condition In February 2024, when she was five, Liliana was diagnosed with Morquio syndrome — also known as mucopolysaccharidosis type IV — a rare genetic condition that ...

The FDA Wednesday approved the first drug to treat people with Prader-Willi syndrome, a rare genetic disease that causes an ...

A new type of cat coat color has officially been discovered. Named salmiak, or ‘salty liquorice,’ the cats have hair strands ...

The treatment of this rare genetic disease is based on successful pre-clinical work of Dr. Antonella De Matteis, Professor of Biology, Department of Molecular Medicine and Medical Biotechnology at ...

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

Johns Hopkins Medicine laboratory scientists say they have developed a potential new way to treat a variety of rare genetic diseases marked by too low levels of specific cellular proteins. To boost ...

The U.S. Food and Drug Administration on Wednesday approved Soleno Therapeutics' drug to treat a rare genetic disorder, ...

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 ...

He passed away on March 1 from POLG Mitochondrial disease, a rare genetic disorder that affects approximately one in 5,000 people. The disease robs the body’s cells of energy, causing ...