Research Funding Opps Seeking Applicants!

The Perelman School of Medicine at the University of Pennsylvania’s Orphan Disease Center (“ODC”) sponsors a Million Dollar Bike Ride each May in Philadelphia. As the result of this year’s successful Million Dollar Bike Ride, the ODC is offering several research funding opportunities in the lysosomal diseases. These grants are open to the international community (they are not limited to University of Pennsylvania or the Children’s Hospital of Philadelphia). These lysosomal disease research funding opportunities are:

1. For mucolipidosis type IV (ML4), one $45,000 pilot grant available to investigators conducting research on all aspects of disease including disease pathogenesis and clinical studies. This grant is made possible by Team Cure ML4, Pedal4Paul, Climb4Carin, and the ML4 Foundation.

2. For the MPS diseases, two $52,000 pilot grants available. ODC is seeking applications directed to treating the central nervous system manifestations of these diseases. These grants are made possible by Team MPS, the National MPS Society and The Ryan Foundation.

3. For Niemann-Pick disease type C (NPC), two $51,000 pilot grants are available. ODC will give preference to research projects developing new therapies for NPC, and translational research projects that improve our understanding of the biology, pathogenesis and disease state (i.e., biomarkers or functional outcome measures to assess therapeutic impact). This grant is made possible by Team NPC, the Andrew Coppola FoundationJammin’ for JPChase the Cure and iPedal4Chad.

4. For Tay-Sachs, Sandhoff, GM-1 or Canavan disease, one $42,000 pilot grant is available focusing on forms of Tay-Sachs, Sandhoff, GM-1, or Canavan disease. ODC is soliciting proposals for innovative research projects that involve basic research, translational studies or clinical studies relevant to the diseases mentioned above. Projects may be focused on (1) pre-clinical and clinical research needs, such as clinical outcome measures, registries, animal models, or biomarkers; or (2) technology approaches such as stem cells, molecular chaperones, substrate inhibitors, small molecule drug screening, gene therapy, novel drug delivery to the brain. This grant is made possible by Team NTSAD and the National Tay-Sachs & Allied Diseases Association.

Eligibility for these funding opportunities is restricted to individuals holding a faculty-level appointment at an academic institution, or a senior scientific position at a non-profit institution or foundation.

Deadline to Apply: Submit your Letter of Interest (“LOI”) in the required online form no later than Friday, September 16, 2016 by 8 p.m. EST. Full application is by invitation only, after review of LOIs.

For the downloadable Million Dollar Bike Ride RFA Guidelines, the link to the online Letter of Interest submission form that must be used, the full list of all available Million Dollar Bike Ride funding opportunities (this includes many non-lysosomal diseases) and other information, visit: current Orphan Disease Center funding opportunities and read the entry entitled “2016 Million Dollar Bike Ride Grants.”

Questions regarding the scientific content of potential projects can be directed to Ms. Molloy at moniquek@nullexchange.upenn.edu; administrative queries should be sent to Ms. Charleston at scharle@nullupenn.edu.

Leukodystrophy Research Videos!

The Global Leukodystrophy Initiative (“GLIA”), in collaboration with the Moser Center for Leukodystophies, held a 3-day conference entitled “Emerging and Translational Biology of Leukodystrophies” at the Kennedy Krieger Institute in Baltimore, Maryland during May 2016. GLIA has now made many scientific-presentation videos freely available from each day of the conference. Visit GLIA’s Conference videos page, where a wealth of recent leukodystrophy scientific research is available for your education and enjoyment. Thank you GLIA for this wonderful opportunity to learn about recent progress affecting newborn screening, treatment and cure for the leukodystrophies!

The Lysosomal Disease Network wishes to express its thanks to Dean and Teryn Suhr of the MLD Foundation, a member of GLIA and one of the Lysosomal Disease Network’s affiliated patient advocacy groups, who provided audiovisual support to GLIA throughout the Conference. Thanks to their tireless efforts, dozens of people from around the world were able to stream the presentations live via the GLIA website.

Free Booklets Pertinent to Rare Disease Patients & Caregivers

The American Health Lawyers Association (AHLA) offers a variety of free booklets that provide education about matters of interest to rare disease patients, their caregivers and family members. For example, these are just a few of the booklets available: “A Handbook for Parents: The Individuals with Disabilities Education Act”“Guide to Legal Issues in Life-Limiting Conditions, 2nd Edition”, and “Guidebook for People with Disabilities”. These free booklets are all part of AHLA’s extensive Public Interest Series. Scroll down to the paragraph entitled Health Care Consumer.

The American Health Lawyers Association, with 13,000 members, is the nation’s largest, nonpartisan, 501(c)(3) non-profit educational organization devoted to legal issues in the health care field.

Sanfilippo Children’s FDN in Australia

The Sanfilippo Children’s Foundation in Australia is currently calling for “Expressions of Interest” for their 2016 Project Grant Round. This opportunity is not limited to researchers in Australia. These grants include translational research grants which provide funding for projects with significant potential to move through the translational pipeline towards treatment of Sanfilippo syndrome. This includes pre-clinical and clinical research to develop new treatments.

These grants also include incubator grants which focus on small-scale projects investigating bright new ideas in treatment of Sanfilippo syndrome. These small-scale projects should have the potential to generate the kind of results that can attract larger-scale funding.

Deadline for the submission of expressions of interest is April 29, 2016. Successful expressions of interest will be invited to submit a full application in mid-May. Deadline for full applications is June 25, 2016. Funding decisions will be made in August 2016. Visit their Web site: Click here for details.