Dana’s Angels Research Trust (DART), the nonprofit organization dedicated to funding medical research, education and treatments for the rare childhood disease Niemann-Pick type C (NPC), often referred to as “childhood Alzheimer’s,” will host its 9th annual DART to the Finish Charity Walk on Saturday, Oct. 3, at Greenwich Point Park in Old Greenwich.
The family-friendly 2-mile walk starts at 9:00am and brings together families, friends and supporters of all ages to raise critical funds for NPC research and treatments.
The Marella family, Philip, Andrea, Andrew and Phil at the 2025 DART to the Finish event in Old Greenwich. Photo: Jeni Howard
Registration is $30 for ages 22 and older; $15 for ages 10 to 21; and free for children under 10. Virtual walker registration is $30. To register, visit dartevents.org .
Last year, more than 200 supporters participated in the DART to the Finish Charity Walk, raising more than $160,000 for NPC research. Funds raised by DART continue to support research ranging from disease tracking biomarkers and newborn screening to new drug development and a potential gene therapy trial.
“This year’s walk comes at an especially promising time for NPC research,” noted Phil Marella, co-founder of DART. “We continues to fund biomarker research alongside other NPC organizations, including the Hide & Seek Foundation for Lysosomal Disease Research and the Ara Parseghian Medical Research Fund.
Biomarkers can help researchers measure how NPC is progressing and whether a treatment is working — which is particularly important in a rare disease where clinical trials involve relatively small numbers of patients. ”
Another major area of progress is gene therapy. Researchers at the National Institutes of Health are working with New York-based biotechnology company Apertura Gene Therapy on a next-generation gene therapy approach for NPC1. The collaboration is testing a new delivery system designed to cross the blood-brain barrier and deliver a therapeutic gene more broadly throughout the central nervous system. The immediate focus for DART is providing a much-needed researcher at the NIH for the gene therapy trial, which is expected to cost approximately $85,000 per year for two years. DART views the project as an important opportunity for the NPC nonprofit community to support collectively. If the preclinical work is successful, DART expects the gene therapy program to move toward a Phase I clinical trial at NIH relatively quickly.
For DART co-founders Andrea and Phil Marella, the advances are deeply personal. They founded DART in 2002 after two of their four children, Dana and Andrew, were diagnosed with NPC. When Dana was diagnosed at age 8, there were no known treatments for the disease. NPC progressively affected her ability to walk, talk and eat, as well as her cognitive abilities. Dana sadly passed away in 2013, just days before her 20th birthday.
For the Marella’s son Andrew, at age 27 today, he is doing considerably well considering he is benefiting from a combination of treatments that did not exist when his sister was diagnosed. His regimen includes the two medications approved by the FDA in 2024 specifically for NPC — Miplyffa (arimoclomol) and Aqneursa (levacetylleucine) — as well as miglustat and monthly spinal taps to receive adrabetadex, also known as cyclodextrin. Miplyffa is FDA-approved for use in combination with miglustat, whileAqneursa is approved to treat neurological symptoms associated with NPC.
“Adrabetadex represents another potentially significant milestone,” shared Phil Marella. “The investigational treatment is currently under FDA Priority Review for infantile-onset NPC, with a target action date of Nov. 17, 2026. Andrew has received this therapy for years through spinal taps and we credit it with helping keep his disease at bay. While having multiple treatment options is remarkable progress, keeping Andrew on a complex four-medicine regimen is also challenging — reinforcing the need to continue developing treatments that are more effective and easier for patients and families to manage.”
Researchers are also advancing nizubaglustat, an investigational oral treatment that Phil Marella hopes will be an improvement on the benefits of miglustat.
Nizubaglustat is a brain-penetrant small-molecule drug similar to Miglustat with a dual mechanism of action and is now being studied in an ongoing Phase 3 clinical trial for NPC. Phase 2 research showed a favorable safety profile and supported moving the drug into the larger Phase 3 study.
“While Andrew and many of the other NPC children are doing so much better today with these treatments, there is still more research that needs to be funded,” said Andrew’s mother Andrea Marella, also co-founder of DART. “This is why we keep walking; why we keep fundraising. The dollars raised means that a child may live longer or have a better quality of life than what was available just a few years ago. Giving our family and families like ours more quality time, is the biggest gift of all.”
DART also continues to support newborn screening efforts to identify babies with NPC earlier, when treatment may have the greatest potential impact. Researchers involved in NPC research will attend this year’s DART to the Finish Charity Walk to speak about their research, new treatments and the progress being made toward better therapies.
“We are deeply grateful to everyone who walks, donates and volunteers, as well as our wonderful sponsors,” said Andrea Marella. “The money raised helps 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 friends and whole community.”
A few weeks before the DART to the Finish charity walk, DART will participate in the 15th annual Health Wellness & Lifestyle Expo 2026 on Saturday, Sept. 12, from 10:00am to 5:00pm at the Harbor Point Boardwalk in Stamford.
DART representatives will be on-site to share information about NPC and the organization’s research efforts and to sign up walkers for the Oct. 3 DART to the Finish Charity Walk.
The free community expo will feature more than 130 sponsors and exhibitors along the Harbor Point waterfront.
Since its founding, DART has raised more than $6.6 million for NPC research, helping support work ranging from laboratory research and biomarker development to clinical trials, drug development and early detection.
To register for the 9th annual DART to the Finish Charity Walk on Saturday, Oct. 3, visit dartevents.org .
To learn more about Dana’s Angels Research Trust, visit danasangels.org , follow DART on Facebook at @danasangels and Instagram at @danasangelsNPC .