Million Dollar Bike Ride

The Penn Medicine Orphan Disease Center (ODC) will be hosting the 5th Annual Philadelphia‐based bicycle ride on Sunday, May 20, 2018 to raise much-needed funds for research in rare diseases.  Team MPS is registered for participation in this event, and registration for cyclists will be announced at a later date. One major announcement to note is … Continued

Board of Directors Election Results

The results are in!  Congratulations to our new board members and those re-elected into their role as Board of Director of the National MPS Society for 2018-2019. We would like to thank all of our board members for their continued commitment to excellence and persistence in this fight to find treatments and cures for all those … Continued

Financial help available for MPS VII patients

The Assistance Fund, an independent charitable patient assistance foundation that helps patients and families facing high medical out-of-pocket costs, has launched a financial support initiative for MPS VII patients and their families. The program offers funds that can be used for copayments, health insurance premiums and incidental medical expenses. “Individuals with MPS VII face a … Continued

FDA approves MPS VII drug

The U.S. Food and Drug Administration today approved Mepsevii (vestronidase alfa-vjbk) to treat pediatric and adult patients with MPS VII, Sly syndrome. The safety and efficacy of Mepsevii, developed by Ultragenyx, were established in clinical trial and were tested on 23 patients ranging from 5 months to 25 years of age. Patients received treatment with … Continued

Sangamo treats first patient with gene therapy

Sangamo Therapeutics treated its first patient in the Phase 1/2 clinical trial (“the CHAMPIONS study“) evaluating SB-913, an investigational in vivo genome editing therapy for people with MPS II, Hunter syndrome. Sangamo aims to treat MPS II by using genome editing to insert a corrective gene into a precise location in the DNA of liver … Continued

Society awards MPS and ML research grants

The National MPS Society’s annual research program has awarded $405,000 in funding for additional research grants. Dr. Sharon Byers – $90,000 general grant over two years SA Pathology (WHC site) Adelaide, Australia “Can the cell cycle be re-set to normal in the MPS growth plate chondrocytes?” Dr. Igor Nestrasil – $60,000 MPS I grant Department … Continued

National MPS Society names interim CEO

Terri Klein, National MPS Society’s director of development and operations, has assumed the title of interim CEO. Terri’s long history with the National MPS Society and her dedication to funding MPS and ML research make her the ideal choice to assume the president’s responsibilities. The National MPS Society board of directors expresses gratitude to outgoing … Continued

Society joins Sanfilippo foundations to support gene therapy development

Abeona Therapeutics Inc., a leading clinical-stage biopharmaceutical company focused on developing novel gene and cell therapies for life-threatening rare diseases, announced today a collaborative agreement between nine Sanfilippo foundations to provide approximately $13.85 million of grants to Abeona in installments for the advancement of the Company’s clinical stage gene therapies for Sanfilippo Syndrome Type A … Continued

Foundation offers assistance for Hunter Syndrome medical expenses

We know that medical expenses are always a headache, so we are excited to share some good news on the health care front for children and adults with MPS II! A charitable foundation, the Assistance Fund, added Hunter Syndrome to its list of covered diseases and established the Hunter Syndrome Financial Assistance Program. The foundation … Continued

Lysogene establishes MPS III parent advisory board

Lysogene MPS IIIA parent advisory board holds inuagural meeting. Each member is a parent of a child with #Sanfilippo and will help the company establish realistic expectations for therapies, hear about clinical trial design and recruitment effort and reiterate the importance of addressing the cognitive problems of those affected by MPS IIIA. “Lysogene is dedicated … Continued