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I am raising funds to save my and Laura's precious sons, Marco and Alexander. These courageous boys are both fighting a very rare and life threatening type of Muscular Dystrophy called BAG3 MFM6 myopathy. This condition is caused by a spontaneous genetic mutation -- the parents and family do not carry it. If you do a quick Google search, you will not even find a Wikipedia page on the disease and what it is about.
Marco, 22-years old now, is confined to a wheelchair, unable to stand, and needs a breathing machine. Alexander will likely suffer the same fate. Both boys have lived with no hope, and the knowledge that their condition is so rare that there is little or no incentive for medical research to be conducted.
Marco's Story
Marco is a beautiful, resilient soul who flew into my life, and grew to be charming child. But as the years passed, Marco's wings got clipped. At 4 he could not sit cross legged on the floor, or grip the ropes of the swing set. He played soccer with AYSO teams until he couldn't run anymore . He played in jazz band until he couldn't hold up a guitar anymore. He swam till his breath began to fail him. But he valiantly kept trying to keep up with his peers.
We went through leg braces, scooter, manual wheelchair, then ventilator and power wheelchair, all within a few years. In 2012 Marco was finally diagnosed with a recently discovered mutation. It was scary - a rare, severe, and rapidly progressing form of muscular dystrophy myopathy. No treatment exists. No one is even trying for a treatment. I had almost given up hope until recently.
Cutting Edge New Research and Partnership with Alexander's Family
Laura, Alexander's mother found us through a YouTube video I had posted during Marco's high school years, and we have partnered together for our common cause.
Dr. Willis contacted us from the University of North Carolina and is working on a drug therapy to reverse the effects of this disease. Dr. Lee at Harvard has done amazing work with gene therapy on a similar disease. We are actively talking to them and other researchers.
Alexander, left, Marco with caretaker Donnetta, right
The therapies are innovative and exciting, but carry risk and drug-companies and funding agencies are reluctant to invest in a rare disease with a small patient population at this stage. We, the two families, are raising money for researchers to kickstart a project specifically designed for our disease, our sons, and other kids with Bag3MFM6. We have a commitment from a researcher for a minimum of a 1 year project. If successful, this will open up doors to therapies for similar diseases, and funding for more research will start flowing.
We want to treat Marco under the Compassionate Use FDA Act . We want to stop Alexander from declining to the state that Marco is now in.
Marco and Quinn
Most children with this mutation have died in their teens or twenties. You can help Marco and Alexander to celebrate more birthdays, fight on, and pave the way for the next generation. We CAN repair their broken wings. Please join us and remember that no amount is too small to donate . Please spread our message. Your support is greatly appreciated!
Thanks a lot, Maneesha Prakash (Marco's mother)
Marco, 22-years old now, is confined to a wheelchair, unable to stand, and needs a breathing machine. Alexander will likely suffer the same fate. Both boys have lived with no hope, and the knowledge that their condition is so rare that there is little or no incentive for medical research to be conducted.
Marco's Story
Marco is a beautiful, resilient soul who flew into my life, and grew to be charming child. But as the years passed, Marco's wings got clipped. At 4 he could not sit cross legged on the floor, or grip the ropes of the swing set. He played soccer with AYSO teams until he couldn't run anymore . He played in jazz band until he couldn't hold up a guitar anymore. He swam till his breath began to fail him. But he valiantly kept trying to keep up with his peers.
We went through leg braces, scooter, manual wheelchair, then ventilator and power wheelchair, all within a few years. In 2012 Marco was finally diagnosed with a recently discovered mutation. It was scary - a rare, severe, and rapidly progressing form of muscular dystrophy myopathy. No treatment exists. No one is even trying for a treatment. I had almost given up hope until recently.
Cutting Edge New Research and Partnership with Alexander's Family
Laura, Alexander's mother found us through a YouTube video I had posted during Marco's high school years, and we have partnered together for our common cause.
Dr. Willis contacted us from the University of North Carolina and is working on a drug therapy to reverse the effects of this disease. Dr. Lee at Harvard has done amazing work with gene therapy on a similar disease. We are actively talking to them and other researchers.
Alexander, left, Marco with caretaker Donnetta, rightThe therapies are innovative and exciting, but carry risk and drug-companies and funding agencies are reluctant to invest in a rare disease with a small patient population at this stage. We, the two families, are raising money for researchers to kickstart a project specifically designed for our disease, our sons, and other kids with Bag3MFM6. We have a commitment from a researcher for a minimum of a 1 year project. If successful, this will open up doors to therapies for similar diseases, and funding for more research will start flowing.
We want to treat Marco under the Compassionate Use FDA Act . We want to stop Alexander from declining to the state that Marco is now in.
Marco and QuinnMost children with this mutation have died in their teens or twenties. You can help Marco and Alexander to celebrate more birthdays, fight on, and pave the way for the next generation. We CAN repair their broken wings. Please join us and remember that no amount is too small to donate . Please spread our message. Your support is greatly appreciated!
Thanks a lot, Maneesha Prakash (Marco's mother)