Joomla gallery extension by joomlashine.com
Main Promo Images
Latest news on Friedreich's Ataxia research
Updated results from clinical trials and research initiatives
Friedreich's Ataxia fundraising activities and staying in touch
Participate and stay informed about our fundraising events. Take part in our Facebook community with FA patients, relatives, researchers and friends from all around the world!
MDA will provide initial seed money of $10 million for MVP's operating capital. Its investments will specifically focus on companies conducting commercially promising research in four disease categories: Duchenne muscular dystrophy (DMD); amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease); spinal muscular atrophy (SMA); and Friedreich's ataxia, myotonic dystrophy, and limb-girdle muscular dystrophy.
MVP will approach the raising, investment and management of funds much like a venture capital firm, utilizing venture capital principles and tools to develop strategies, evaluate each investment opportunity, make quick and soundly based decisions and comprehensively report results against milestones to investors.
MVP will seek donations from a new body of philanthropists -- individuals with both a familial connection to muscle diseases and the financial ability to make a significant contribution ($250,000 or more). MVP will then use the donations to place initial targeted investments into biotechnology companies and academic institutions with highly promising therapies for these diseases. These investments by MVP will not only provide needed funding but will also serve to attract follow-on investments from other sources such as pharmaceutical and biotech companies.
"We're excited about MVP because it will help us to overcome the critical funding gap that can occur in research on promising new drugs and therapies," said MDA President & CEO Gerald C. Weinberg.
MVP's venture capital model addresses the well-known drug development challenge in which promising work on ideas for new drug therapies, development of which can take many years and cost more than $1 billion to bring to fruition, can run out of money and be abandoned. The funding problem is worsened when the drug or the therapy is being developed for a disease that affects a relatively small patient population base, with a relatively small return on investment. MVP overcomes these problems by joining the entrepreneurial spirit of donors with best business practices to drive a specific therapeutic development.
"As a venture philanthropy operating in a very distressed economy and a competitive philanthropy landscape, MVP will hold the companies we invest our donations in fully accountable through milestone-driven contracts," MVP Executive Director Sharon Hesterlee, Ph.D. said. "But we will also hold ourselves accountable to our donors through a structure that enables them to have an advisory role in MVP's investment evaluation process, and to see firsthand that MVP is meeting its goals as an organization."
About MDA: MDA is a voluntary health agency supporting programs of worldwide research, comprehensive services, advocacy, and professional and public health education for muscular dystrophy and related diseases. MDA is the largest nongovernmental sponsor of neuromuscular disease research. The Association's programs are funded almost entirely by individual private contributors. Visit http://www.mda.org for more information.
Muscular Dystrophy Association (MDA)
If you feel like reading an unputdownable novel while collaborating with a just and solidary cause, "The Legacy of Marie Schlau" is your book! 100% of all funds raised will be dedicated to medical research to find a cure for Friedreich's Ataxia, a neurodegenerative disease that affects mostly young people, shortening their life expectancy and confining them to a wheelchair.
The life of Marie Schlau, a German Jewish girl born in 1833 hides great unsolved mysteries: accidents, disappearances, enigmas, unknown diagnoses, disturbing murders, love, tenderness, greed, lies, death ... alternatively a different story unfolds every time and takes us closer to the present. Thus, there are two parallel stories unravelling, each in a different age and place, which surprisingly converge in a revelatory chapter.
Paperback and Kindle versions for "The legacy of Marie Schlau" available for sale at Amazon now!
Currently, BabelFAmily is financing two promising research projects aimed at finding a cure for Friedreich's Ataxia. Whenever you make a donation to us or purchase a copy of "The legacy of Marie Schlau", this is where all funds raised will be devoted to:
1) Gene Therapy for Friedreich's Ataxia research project:
The project is the result of an initiative of Spanish people affected by this rare disease who are grouped in GENEFA in collaboration with the Spanish Federation of Ataxias and the BabelFAmily. The Friedreich’s Ataxia Research Alliance (FARA), one of the main patients’ associations in the United States now joins the endeavour.
2) Frataxin delivery research project:
The associations of patients and families Babel Family and the Asociación Granadina de la Ataxia de Friedreich (ASOGAF) channel 80,000 euros of their donations (50% from each organisation) into a new 18-month project at the Institute for Research in Biomedicine (IRB Barcelona). The project specifically aims to complete a step necessary in order to move towards a future frataxin replacement therapy for the brain, where the reduction of this protein causes the most damage in patients with Friedreich’s Ataxia.
The study is headed by Ernest Giralt, head of the Peptides and Proteins Lab, who has many years of experience and is a recognised expert in peptide chemistry and new systems of through which to delivery drugs to the brain, such as peptide shuttles—molecules that have the capacity to carry the drug across the barrier that surrounds and protects the brain. Since the lab started its relation with these patients’ associations in 2013*, it has been developing another two projects into Friedrich’s Ataxia.