This Is A Custom Widget

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.

This Is A Custom Widget

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.

Monthly Archives: August 2018

//August

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 [...]