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This Sliding Bar can be switched on or off in theme options, and can take any widget you throw at it or even fill it with your custom HTML Code. Its perfect for grabbing the attention of your viewers. Choose between 1, 2, 3 or 4 columns, set the background color, widget divider color, activate transparency, a top border or fully disable it on desktop and mobile.

In The News

/In The News

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 [...]

Symptoma.com – Helping Physicians To Make The Right Diagnosis For Their Patients.

Symptoma.com is a search engine for diseases. Patients and doctors enter symptoms and receive a list of matching causes - sorted by probability. Try it out for MPS related symptoms, such as "middle ear infections, bushy eyebrows": https://www.symptoma.com/en/search?query=middle%20ear%20infections%2C%20bushy%20eyebrows MPS I: https://www.symptoma.com/en/info/mucopolysaccharidosis-1 MPS II: https://www.symptoma.com/en/info/mucopolysaccharidosis-2 MPS III: https://www.symptoma.com/en/info/mucopolysaccharidosis-3 MPS IV: https://www.symptoma.com/en/info/mucopolysaccharidosis-4 MPS VI: https://www.symptoma.com/en/info/maroteaux-lamy-syndrome [...]

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 [...]

Canadian Consensus Position Statement for the Diagnosis and Management of MPS II

June 15, 2018 VANCOUVER, BC – Today, the Canadian MPS Society issued a national consensus statement that provides updated Canadian guidelines for the management of patients with MPS II. A consensus meeting was held in Toronto, Ontario, including a multidisciplinary group of experts in the management of patients with MPS II. The group reviewed available published guidelines [...]

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 [...]

2018 Summer Studentship Research Grants – Request for Applications

The Canadian Society for Mucopolysaccharide & Related Diseases Inc. (The Canadian MPS Society) is once again offering a limited number of Summer Studentship Research Grants for medical students and university undergraduates.  It is our hope that these opportunities may result in more individuals being attracted to research careers involving lysosomal storage disorders. The studentships are tenable [...]