The FDA recently announced a national partnership to accelerate gene therapies for rare diseases, and for families like the Alams, that's a big deal.
The Alam family is coming together with the community to raise money for Children's National to start research.
Over the years, the Alam family has been helping their son Ryan live life to the fullest after he was diagnosed with a terminal neurological disease.
Ryan, a DMV native, was diagnosed with NBIA, a rare neurodegenerative disease several years ago. The type Ryan has, called MPAN, is even rarer - with fewer than 100 known cases worldwide.
Over time, patients lose the ability to walk and swallow while developing dementia and Parkinson’s, and as it stands now, there is no cure.
"He’s dealt with the most horrific adversities that any human being can deal with and he’s met it with a smile," said Ryan’s dad, Faisal Alam.
Ryan and his family began a fundraising mission in 2019 to raise money for research to fight his deadly disorder.
The money donated does not go to his own treatment, but rather to research aimed at helping others who are or will be fighting the same condition. Over $500,000 has been raised so far.
For more information and to donate, click here.