MPS Registry

DURHAM, N.C. — With the click of a mouse, patients with a rare disease will now be one step closer to cures for mucopolysaccharidosis and mucolipidosis.

The National MPS Society, an organization that helps find cures for MPS and related diseases through research, advocacy and awareness, has teamed up with 22 MPS advocacy organizations and PatientCrossroads, a patient-centered data company that optimizes the search for better treatments for diseases.

MPS and ML patients will now be able to use the secure online PatientCrossroads CONNECT platform to contribute, view and share data. The data offer researchers and medical professionals the opportunity to gather information on what it means to live with these diseases and to find patients for treatments, research studies, clinical trials and post-treatment programs.

MPS and ML are genetic lysosomal storage diseases (LSD) caused by the body’s inability to produce specific enzymes. In individuals with MPS and related diseases, the missing or insufficient enzyme prevents cells from recycling properly, resulting in the storage of materials in virtually every cell of the body. This causes progressive damage throughout the body, including the heart, bones, joints, respiratory system and central nervous system.

“ConnectMPS comes at a time when the biopharmaceutical community is facing a lack of patient-reported outcomes and experiences — information that is critical to therapeutic development and eventual drug approval,” said Mark Dant, executive director of the National MPS Society. “By collaborating in this pan-disease program, MPS organizations can demonstrate their commitment to working collaboratively to help locate treatments for MPS and ML.”

“MPS is so rare that little funding was available to invest in the basic science needed to identify drug candidates, so families raised funds to support early research into treatments for our sons and daughters,” said Jill Wood, co-founder of Jonah’s Just Begun, an MPS III advocacy organization. “Hope for new treatments is on the horizon, and now we have the opportunity to build a research-ready community that is dedicated to accelerating the therapeutic development process.”

Any person diagnosed with an MPS-related disorder can register for ConnectMPS, including all forms of MPS and ML. To join, visit www.connectmps.org.

 

About the National MPS Society

The National MPS Society exists to find cures for MPS and ML. We provide hope and support for affected individuals and their families through research, advocacy and awareness of these devastating disorders. MPS and ML are genetic lysosomal storage diseases (LSD) caused by the body’s inability to produce specific enzymes. The malfunctioning cells are unable to recycle waste materials, causing progressive damage throughout the body, including the heart, bones, joints, respiratory system and central nervous system. For information on MPS and ML, please visit www.mpssociety.org.

 

About PatientCrossroads

Patient Crossroads is a patient-centered data company in Silicon Valley with an innovative business model that optimizes the search for better treatments for diseases. We aggregate and curate data to amplify the voice of patients. Our secure, cloud-based, multilingual platform enables users to conduct targeted studies and quantify value during every phase of drug development and commercialization.

We deliver patient insights faster, and for far less, than traditional registries and CRO organizations. Our approach fosters ongoing patient engagement. We make personalized medicine and pan-disease research possible. Since 2007, we have developed programs for more than 400 diseases through our work with over 100 advocacy groups, NIH, PCORI, biotech and pharma companies. For more information, visit http://www.patientcrossroads.com.

 

About Jonah’s Just Begun

Jonah’s Just Begun – Foundation to Cure Sanfilippo Inc. is a 501(c)3. JJB raises funds and then distributes them to academic researchers focused on Sanfilippo Type C. We have two goals: first, to drive the science that will ultimately lead to a cure for Sanfilippo Type C; and second, to raise awareness for all rare diseases. We also empower and encourage others affected by rare diseases to advocate for cures. For more information, visit http://jonahsjustbegun.org.

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