HNF launches golf fundraiser for rare CNTNAP1 gene therapy research

The Hereditary Neuropathy Foundation will host James’ Scramble for a Cure on Aug. 14 at Tijeras Creek Golf Club in Rancho Santa Margarita, California, to raise money for CNTNAP1 gene therapy research. The event centers on 8-year-old James McCarthy, who lives with an extremely rare CNTNAP1 mutation and severe CMT-related complications.

Why it matters: - The fundraiser will support research into CNTNAP1, an ultra-rare genetic disorder with no cure. - Gene therapy is being positioned as the most promising path forward for children and families affected by the condition. - The event links fundraising to a real patient story, which can help drive donations, sponsorships and awareness for a rare disease with limited treatment options.

What happened: - The Hereditary Neuropathy Foundation announced James’ Scramble for a Cure, a charity golf tournament and evening gala set for Friday, Aug. 14, 2026, at Tijeras Creek Golf Club in Rancho Santa Margarita, California. - All proceeds will benefit CNTNAP1 gene therapy research. - Tickets and sponsorship opportunities are available at JamesCure.GiveSmart.com.

The details: - Golf event registration and lunch open at 11:00 a.m. - The putting contest starts at 12:30 p.m. - The shotgun start begins at 1:00 p.m. - The evening program is open to families and begins at 6:00 p.m. with dinner, auction, awards and live entertainment. - A helicopter golf ball drop is scheduled for 6:30 p.m. - The silent auction closes at 7:30 p.m. - James McCarthy is 8 years old and lives with an extremely rare mutation of the CNTNAP1 gene. - James was born unable to breathe, was immediately intubated and spent nearly a month in the NICU at Children’s Hospital of Orange County. - Doctors diagnosed James with bilateral vocal cord paralysis, hypotonia and other challenges. - James underwent tracheostomy and gastrostomy tube surgeries within his first weeks of life. - James now uses a wheelchair and is nonverbal. - James has learned to swallow, move his limbs and smile through therapy. - James recently began communicating using eye-tracking technology. - Over the past year and a half, James’ health has improved and his family says his developmental and social growth has been remarkable. - James’ family said, “We pray for a day that we can hear our sweet boy cry or laugh.” - James’ family also said they hope consistent therapy and research will help him keep reaching new “inch stones.” - CNTNAP1 is a recessive genetic disorder that causes loss of the gene’s protein product, CASPR. - The missing CASPR protein disrupts myelinating cells from nerve cell axons. - The disorder causes profound demyelination and weakness in both the central and peripheral nervous systems. - CNTNAP1 is one of the severe CMT, or Charcot-Marie-Tooth, syndromes. - There is currently no cure. - Researchers have made significant progress and are actively pursuing gene therapy studies for CNTNAP1. - The Hereditary Neuropathy Foundation is a global nonprofit focused on quality of life for people with CMT disease and on accelerating research toward treatments and cures. - HNF says its work includes research initiatives, patient-centered clinical trial readiness programs, regulatory engagement and community education. - HNF says its goal is to bridge science, industry and patient voice to help drive CMT drug development.

Between the lines: - The event mixes a golf outing, family-friendly gala and auction to broaden the donor base beyond golfers. - Centering the fundraiser on James McCarthy gives the campaign a personal narrative that can make an abstract gene-therapy effort more tangible. - The emphasis on “inch stones” reflects a long-term care story where small developmental gains matter.

What’s next: - Organizers are seeking golfers, evening guests, sponsors, donated auction items and direct donations. - The event will raise funds that can support continued CNTNAP1 research efforts. - More information and registration are available at JamesCure.GiveSmart.com.

The bottom line: - James’ Scramble for a Cure turns one family’s rare-disease journey into a fundraising push for research that could move CNTNAP1 closer to treatment options.