**FAer Michael Gehr arriving at Scientia Clinic, site of Design Therapeutics safety trial**

Recently, FAer and Cure FA Soirée co-founder Michael Gehr spent six weeks Down Under participating in a Design Therapeutics open-label, phase 1 safety trial for a new investigational drug designed to address the underlying cause of Friedreich’s ataxia by restoring endogenous frataxin (FXN) protein production to therapeutic levels. During the trial, Michael endured approximately two dozen needle procedures, including four weekly IV infusions, numerous blood draws, and three thigh muscle biopsies. As expected given the short treatment period, he did not experience any noticeable clinical benefits.

Because this was an open-label study, Michael received the investigational drug; however, the primary objective of the trial was to evaluate safety. Neither Michael nor the healthy volunteers and FA participants enrolled before him experienced major adverse effects. In addition to safety monitoring, blood samples and muscle biopsies will be analyzed to determine whether participants produced increased levels of frataxin, which is deficient in individuals with FA. If frataxin production is shown to increase, prolonged treatment could, in theory, at least slow disease progression—and, with some optimism, potentially result in modest functional improvement. Given that the effects of low frataxin accumulate over many years, any positive impact would likewise be expected to emerge gradually over time. Learn more about the drug and the trial in a June 2025 news release.

While the clinical trial was demanding, it was only part of Michael’s experience abroad. Outside the hospital setting, the weeks in Australia also became an opportunity for family, exploration, and connection. Michael’s father, Eric, accompanied him to Australia, and his mother, Liz, joined them four weeks later. Traveling halfway across the globe, the Gehrs made the most of their time abroad. Between Michael’s trial appointments, he and Eric explored both the iconic and lesser-known sights of Sydney and enjoyed the city’s renowned food scene. Along the way, they encountered flora and fauna that might seem exotic to Americans but are commonplace in Australia.

The journey also created opportunities for new adventures, new friendships, and reconnecting with old ones. All three traveled to Brisbane, about 600 miles north of Sydney, for several days to experience another region of Australia and reunite with friends who had lived in Oklahoma more than a decade earlier. After the trial concluded, the Gehrs continued on to New Zealand for a week to visit the family of a woman who had been an exchange student with Liz’s family in the 1980s.

Adventure was a recurring theme throughout the trip. Eric surfed in Australia and later took on spin cycling, mountain biking, paragliding, and bungee jumping in New Zealand. Liz joined in as well, opting for a gentler paragliding experience.

What had been a place of routine, exploration, and joy suddenly became associated with tragedy. Speaking candidly about the Bondi Beach shooting and its emotional impact, Eric said, “We were heartbroken to read about the horrible shooting in Bondi Beach. Michael and I lived there for 5 weeks. We'd been all around the park where the shooting took place, which was also next to the beach where I learned to surf. Our memories are of a happy, relaxed atmosphere, and it seemed to have a live-and-let-live vibe. The only darker indications I noted were the security officers outside the several temples I ran by on Saturday mornings. It's hard to wrap my head around; I look at news coverage and remember being there so recently.

It also felt very strange for Michael and me to be gone for so long, but candidly it was exciting to live somewhere else for a while. “