Mucopolysaccharidosis Type I News and Research

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Novel drug may be the answer in battling resistance in aggressive breast cancer

Novel drug may be the answer in battling resistance in aggressive breast cancer

FDA grants Rare Pediatric Disease Designation for Immusoft’s Iduronicrin genleukocel-T

FDA grants Rare Pediatric Disease Designation for Immusoft’s Iduronicrin genleukocel-T

FDA approves drug to treat pediatric, adult patients with rare Sly syndrome

FDA approves drug to treat pediatric, adult patients with rare Sly syndrome

FDA grants permission to market new Seeker System for screening rare metabolic disorders in newborns

FDA grants permission to market new Seeker System for screening rare metabolic disorders in newborns

Researchers agree to test new stem cell gene therapy for Sanfilippo disease in human trial

Researchers agree to test new stem cell gene therapy for Sanfilippo disease in human trial

FDA clears Sangamo BioSciences' SB-318 IND application for treatment of MPS I

FDA clears Sangamo BioSciences' SB-318 IND application for treatment of MPS I

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

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

Research finding opens door to potential treatment for MPS IIIB

Research finding opens door to potential treatment for MPS IIIB

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

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

Gene therapy developed by UAB cures pediatric Sanfilippo Syndrome A in animal models

Gene therapy developed by UAB cures pediatric Sanfilippo Syndrome A in animal models

EMA grants orphan designations to Ultragenyx UX001, UX003 for treatment of HIBM and MPS 7

EMA grants orphan designations to Ultragenyx UX001, UX003 for treatment of HIBM and MPS 7

BioMarin commences GALNS Phase 3 trial in patients with Morquio A Syndrome

BioMarin commences GALNS Phase 3 trial in patients with Morquio A Syndrome

New study offers hope for children with MPS I genetic disorder

New study offers hope for children with MPS I genetic disorder

Lentigen receives NIH STTR grant for Hunter syndrome lentiviral gene therapy

Lentigen receives NIH STTR grant for Hunter syndrome lentiviral gene therapy

BioMarin announces update on BMN 110 Phase I/II trial for Morquio A Syndrome

BioMarin announces update on BMN 110 Phase I/II trial for Morquio A Syndrome

One injection of gene therapy spreads through brain in animal study

One injection of gene therapy spreads through brain in animal study

First treatment for Hunter disease

First treatment for Hunter disease