Mucopolysaccharidosis Type II News and Research

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Dying patients with rare diseases struggle to get experimental therapies

At 15, Autumn Fuernisen is dying. She was diagnosed at age 11 with a rare degenerative brain disorder that has no known cure or way to slow it down: juvenile-onset Huntington's disease.

14 Jul 2021

FDA clears ArmaGen's AGT-182 IND application for treatment of Hunter syndrome

ArmaGen, Inc., a privately held biotechnology company focused on developing novel therapies to treat severe neurological disorders, announced today that the Investigational New Drug (IND) application for the company's lead product candidate, AGT-182 for the treatment of Hunter syndrome, has been accepted by the U.S. Food and Drug Administration and is now active.

11 Dec 2014

FDA grants orphan drug designation to ARMAGEN's AGT-182 for treatment of Hunter syndrome

ARMAGEN announced today that the U.S. Food and Drug Administration has granted orphan drug designation to its lead product AGT-182 for the treatment of mucopolysaccharidosis type II (also known as Hunter syndrome or MPS II).

19 Jul 2013

Lentigen receives NIH STTR grant for Hunter syndrome lentiviral gene therapy

Lentigen Corporation, a biotechnology company specializing in the development and manufacture of lentiviral gene delivery technologies, announced today that it has received a National Institutes of Health (NIH) small business technology transfer (STTR) grant for a program on "Lentiviral Gene Therapy for Mucopolysaccharidosis."

19 May 2010

First treatment for Hunter disease

An article reporting results from a pivotal clinical trial that helped gain U.S. Food and Drug Administration approval for the first treatment for Hunter disease has been published online by the journal Genetics in Medicine.

16 Aug 2006