Meet Aubrie
This is our friend Aubrie. She is 7 years old and loves to swim, dance, and do karate. Aubrie has a very rare mitochondrial condition called AARS2 mutation. Her mom co-founded CureARS to fund research for treatment and a cure. You can help Aubrie by donating to CureARS or by purchasing the book, Chondrie, that her mom wrote to help teach kids about mitochondria.
Meet Hope
This is our friend Hope. She is 4 years old and loves to sing and listen to Taylor Swift. Hope has a very rare mitochondrial condition called MLS syndrome. We met Hope and her mom and aunt while advocating for elamipretide approval. They are our elamipretide sisters! Hope's family worked tirelessly to make sure that elamipretide would get approved for Hope, Gilby, and everyone who needs it. Her family also runs a non-profit called Hope for Hall that builds inclusive playgrounds in Hall County, Georgia. They even got the Georgia state legislature to pass a law in May of 2025 that requires future elementary school playgrounds be built with inclusivity in mind! You can help Hope by donating to Hope for Hall and learning more about the importance of inclusivity in playgrounds.
Meet Isaac
This is our friend Isaac. He is 19 months old and he is a liver transplant warrior. Isaac has a condition called OTC deficiency which affects his liver's ability to breakdown ammonia. He had a liver transplant at 4 months old, but now he needs a new liver. He is looking for a match from a living donor. His family is sharing his story in hopes that someone will be his perfect match! You can help Isaac by sharing his story to help find a match and purchasing an #IsaacStrong shirt to spread awareness.
Meet Loic
This is our friend Loic. He is 4 years old and he loves red, white, and blue and making his family laugh. Loic has a very rare condition called PKAN. His mom started the Loving Loic Foundation to find a cure for PKAN. They are raising funds for a gene therapy that is currently in development. You can help Loic by donating to the Loving Loic Foundation or helping to support fundraising through in-kind donations and vendor partnerships.
Meet Lottie
This is our friend Lottie. She is (almost!) 5 years old and is an absolute sweetheart! Lottie was born with Down syndrome and her parents recently found out that she also has Sanfilippo syndrome, which is a rare neurodegenerative condition. Lottie is waiting on FDA approval for a gene therapy called UX-111, which will help to stop the progression of Sanfilippo syndrome. You can help Lottie by visiting ApproveHopeNow.com and contacting your congressional leaders and the FDA to act with urgency in approving this therapy. You can also donate to the Cure Sanfilippo Foundation and buy Lottie gear.
Meet Madeline
This is our friend Madeline. She is 9 months old and beyond adorable. When Madeline was 6 months old, her parents found out she has a very rare condition called PKAN. They are working to raise funds for a gene therapy that is currently in development. You can help Madeline by donating to the Loving Loic Foundation or helping to support fundraising through in-kind donations and vendor partnerships. Madeline is also competing to be Baby2Baby's 2025 Baby of the Year, you can vote for her daily!
Meet Noah
This is our friend Noah. He is 4 years old and an absolute fighter as he has survived four cardiac arrests. We met Noah's mom on instagram and have been learning all about Noah's condition and the non-profit that his mom helped start called Heart of PPA2. They are working to raise funds for research to find a cure. You can help Noah by donating to research or helping to support fundraising through in-kind donations and vendor partnerships.
Meet Sadie Rae
This is our friend Sadie Rae. She is 9 years old and she has the most incredible personality full of light and sass! Sadie has Sanfilippo syndrome, which is a rare neurodegenerative condition. We met Sadie's mom and aunt through our advocacy efforts to get elamipretide approved. Sadie is waiting on FDA approval for a gene therapy called UX-111. You can help Sadie by visiting ApproveHopeNow.com and contacting your congressional leaders and the FDA to act with urgency in approving this therapy.
Meet Violet
This is our friend Violet. She is a sweet 4 year old with a rare condition called PDCD. This mitochondrial condition can cause physical and neurological disabilities because of the body's impaired ability to metabolize carbohydrates and a resulting build up of lactic acid. Violet and her PDCD friends need FDA approval for a promising treatment called DCA. You can help Violet by visiting ApproveDCA.com and contacting your congressional leaders and the FDA to act with urgency in approving this treatment.
