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Research and Therapies

/Research and Therapies

RGX-121 for the Treatment of Mucopolysaccharidosis Type II (MPS II)

November 7, 2018 - REGENXBIO announced that the first subject was dosed at the Children's Hospital of Pittsburgh by a medical team led by Dr. Maria Escolar, Associate Professor of Pediatrics, Director of Program for the Study of Neurodevelopment in Rare Disorders at the Children's Hospital of Pittsburgh and primary investigator for the Phase I/II clinical [...]

Sangamo Announces 16 Week Clinical Results Including Reductions In Glycosaminoglycans In Phase 1/2 Trial Evaluating SB-913, A Zinc Finger Nuclease Genome Editing Treatment For MPS II (Hunter Syndrome)

RICHMOND, Calif., Sept. 5, 2018 /PRNewswire/ -- Sangamo Therapeutics, Inc. (NASDAQ: SGMO) today reported 16 week reductions in urinary glycosaminoglycans (GAGs), a key biomarker of Mucopolysaccharidosis Type II (MPS II) disease pathophysiology, in Cohort 2 of the Phase 1/2 CHAMPIONS Study evaluating SB-913.  SB-913 is a zinc finger nuclease (ZFN) in vivo genome editing product candidate being [...]

Mucopolysaccharidosis Type I (MPS I) Pilot Grant Program. One-year grants up to $150,000 (direct costs) will be available to develop novel nucleic and cell based treatments for MPS I.

The Orphan Disease Center (ODC) at the University of Pennsylvania is pleased to announce the Mucopolysaccharidosis Type I (MPS I) Pilot Grant Program. One-year grants up to $150,000 (direct costs) will be available to develop novel nucleic and cell based treatments for MPS I. The proposed therapeutic strategies should address the CNS manifestations of MPS I in patients with the severe [...]

Lysogene MPS IIIA Clinical Trial Update

MPS IIIA is predominantly a central nervous system disease causing cognitive disability, progressive loss of acquired skills, behavioral and sleep disturbance. LYS-SAF302 is a gene therapy intended to deliver a functional copy of the SGSH gene to the brain. This is a phase 2-3 study to assess the efficacy in improving or stabilizing the neurodevelopmental state [...]

Morquio B patient/caregiver survey: First insight into the natural course of a rare GLB1 related condition

Morquio B disease (MBD) or Mucopolysaccharidosis type IV B (MPS IV B) is caused by particular GLB1 mutations specifically affecting the affinity of beta-galactosidase to keratan sulphate, resulting in dysostosis multiplex resembling Morquio A (MPS IV A) disease (GALNS deficiency). Additional neuronopathic features of GM1 II/III (juvenile/adult) gangliosidosis have been reported in some patients. The patient/caregiver online survey was aimed at [...]

Regenxbio receives FDA fast track designation for RGX-111 gene therapy for the treatment of MPS I

June 12, 2018 at 5:36 PM EDT - Novel, one-time, direct-to-CNS investigational treatment for MPS I designed to prevent the progression of cognitive deficits- Phase I clinical trial expected to enroll children and adults with MPS I- Expect to initiate patient recruitment and dosing in mid-2018 ROCKVILLE, Md., June 12, 2018 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX), [...]

Clinical trial for genome editing treatment in MPS I and MPS II announced

"Patients with MPS I and MPS II have very few treatment options, and we are excited to expand access to our clinical trials to the U.K.," said Dr. Edward Conner, Chief Medical Officer at Sangamo. "We are pleased with the MHRA's rapid action on our CTA applications and to be working closely with them to advance [...]

Regenxbio receives FDA fast track designation for RGX-121 gene therapy for the treatment of MPS II

May 2, 2018 at 7:00 AM EDT -- Novel, one-time, direct-to-CNS investigational treatment for MPS II designed to prevent the progression of cognitive deficits-- Phase I/II clinical trial expected to enroll children with MPS II-- Expect to initiate patient recruitment and dosing in mid-2018 ROCKVILLE, Md., May 2, 2018 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX), a [...]

ArmaGen Update on Hurler Syndrome Development Program

AGT-181: Investigational Therapy for the Treatment of Patients with Hurler Syndrome AGT-181 is an investigational enzyme replacement therapy designed to treat both the body-related and central nervous system-related symptoms and complications of MPS I. Currently approved treatments for MPS I are unable to penetrate the blood-brain barrier (BBB), a filter that protects the brain from harmful substances like toxins and bacteria but allows [...]