DART to the Finish Walk Raises $160K for Rare Childhood Disease

Dana’s Angels Research Trust (DART), the nonprofit organization dedicated to funding medical research, education and treatment of the rare childhood disease Niemann-Pick Type C (NPC), often referred to as “childhood Alzheimer’s,” had the most perfect fall day for its 8th annual DART to the Finish Charity Walk, held Saturday, October 4, at Greenwich Point Park. The event drew more than 200 supporters of all ages to walk 2-miles and help raise funds for critical research into new treatments that supports children and young adults with NPC. The event was one of the most successful walks for DART, raising more than $160,000 for NPC research.

“We are deeply grateful to everyone who walked, donated and volunteered, as well as our wonderful sponsors,” said Andrea Marella, DART co-founder. “The money raised will immediately help accelerate NPC research and care, supporting clinical trials, expanding newborn screening and advancing access to approved and emerging treatments. We couldn’t do the important work we do without the amazing support of our family, friends and whole community.”

Andrea and her husband, Phil, founded DART in 2002 after two of their four children, Dana (1993–2013) and Andrew, 26, were diagnosed with NPC. “When Dana was diagnosed at age eight, there were no known treatments for NPC. The disease rapidly impacted her ability to walk, talk, eat and affected her cognitively. Dana sadly passed away just days before her 20th birthday,” noted Phil Marella. “Our son Andrew was just five when he was diagnosed and started treatment. Through research that DART helped fund, we were able to start him — and other children impacted by NPC — with medications. Andrew, who is 26 today, and so many other NPC kids are alive today because of the important NPC and rare-disease work that DART and partner organizations have funded.” Phil noted that Andrew still experiences the detrimental impact of NPC, but is still able to work part time at The Prospector Theater in Ridgefield, Connecticut, and participates in family life and with friends. “Your generosity helps make that possible,” said Phil Marella.

NPC is a genetic cholesterol-storage disorder that causes progressive neurological decline affecting movement, speech and cognition. For many years there were no approved treatments. Thanks in part to two decades of DART-supported research, the Food and Drug Administration has approved three NPC medications — including in 2024 Miplyffa (in combination with Miglustat) and Aqneursa, and a fourth medication is expected to be approved in the next year. These treatments help the 900 children around the world diagnosed with NPC. This summer and fall, Andrew was able to add FDA-approved Aqneursa and Miplyffa to his regimen. He has undergone more than 180 spinal taps to receive Adrabetadex (cyclodextrin), another therapy DART has helped support through research and clinical trials. These medicines and treatments are here today in part because of DART-backed efforts, along with other NPC-focused nonprofits which are helping to expand options for patients and families.

DART’s funding spans the bench to the bedside. At the National Institutes of Health, DART supports scientist Dr. Antony Cougnoux in Dr. Forbes “Denny” Porter’s lab, where researchers are mapping how NPC disrupts cholesterol handling in the brain to guide next-generation strategies, including gene therapy. DART also helped launch SOAR-NPC, a collaborative drug-development program, and funded the first-of-its-kind newborn-screening now active in eight New York hospitals, which identifies affected infants earlier, when intervention can have the most impact.

At the DART to the the Finish Charity Walk, special guest speaker was Dr. Cristin Davidson, a scientist with the National Institute of Health’s Eunice Kennedy Shriver National Institute of Child Health and Human Development and researcher with SOAR-NPC. Dr. Davidson shared that there is a lot of impactful research happening, including work into bio-markers, which shows if disease is progressing or medication is working, with some of the data coming from Parkinson’s disease; small molecule research is happening as a collaboration with University of Illinois, University of Michigan and University of Iowa, which could forward other medication usage for NPC; the third area is gene therapy, which is a big effort at NIH in collaboration with the University of Florida, where they are getting close to a Phase I clinical trial; the fourth exciting news is that the drug Adrabetadex has had clinical trial data showing that infants given this medication have a lower mortality rate than those who haven’t been treated. “Normally the lifespan is just a few years for an infant diagnosed with NPC and this medication is changing that — and that is really exciting,” noted Dr. Davidson.

The science of NPC is also informing research into more common conditions. Because cholesterol storage is a shared pathway, NPC discoveries are helping point to potential treatments for Alzheimer’s, Parkinson’s, ALS and cardiovascular diseases.

Since its founding 23 years ago, DART has raised more than $6.6 million for NPC research. DART also thanked event sponsors and community partners, including Landis Partners, the Weisenburger Trust, Trinity Church, Corient Private Wealth LLC, Jusko Wealth Management Group, The Huston Family, The Charles A. Mastronardi Foundation, the Prospector Theater, The Jones Family, Palmer Point Marina, Manfredi Jewels, Greenwich Shell, Splash Car Wash, Gault Family Companies, Costco, and Greenwich Emergency Medical Services, Inc. continued support.

To learn more about Dana’s Angels Research Trust (DART) or to donate, visit danasangels.org