Weekly Spotlight - 31.10.24

In the News

Vatiquinone Nears FDA Approval for Friedreich's Ataxia Treatment

PTC Therapeutics plans to seek FDA approval for vatiquinone, an oral therapy for Friedreich's ataxia (FA), by the end of the year. This decision follows promising results from the MOVE-FA trial, which tested the drug in both children and adults. Although the primary goal of slowing overall disease progression wasn't met, vatiquinone showed significant benefits in improving upright stability, a crucial factor for younger patients. The extension study further supports vatiquinone's potential, showing a 50% slower decline in disease progression compared to untreated patients. With a strong safety profile and no serious side effects, vatiquinone could become a vital treatment option, especially for children and adolescents with FA. The Friedreich's Ataxia Research Alliance welcomes this development, highlighting the urgent need for effective therapies.

Wearable Sensors Enhance Monitoring of Friedreich's Ataxia Severity

Friedreich's Ataxia (FRDA) is a challenging condition affecting movement and coordination. Traditional assessments are often subjective and limited. This study introduces wearable sensors for at-home monitoring, offering a more comprehensive view of patients' daily lives. By tracking physical activity and arm movements, these devices provide valuable insights into disease severity, potentially improving patient care and treatment outcomes. The study involved 39 participants, using wearable sensors for a week. Results showed significant correlations between sensor data and clinical measures, highlighting the potential of these devices in real-world monitoring. Machine learning models further enhanced predictions of disease severity. This approach promises a more accurate understanding of FRDA, supporting better therapeutic interventions and improving quality of life for patients and their families.

Promising New Treatment for Friedreich's Ataxia Shows Positive Results

Larimar Therapeutics is making strides in treating Friedreich's Ataxia with their promising drug candidate, nomlabofusp (CTI-1601). This innovative treatment aims to correct frataxin deficiency, a key issue in the condition. The drug's design allows it to penetrate cells and deliver functional frataxin protein to mitochondria, potentially improving patient outcomes. The ongoing studies, including a phase 1 trial and an open-label extension study, show encouraging results. The phase 1 trial involved 55 patients and focused on safety and tolerability, while the extension study continues to explore efficacy and long-term effects. Larimar plans to seek accelerated approval, offering hope to patients and families affected by this challenging condition.

Power Chair Reflections: Navigating Life Without It

Jean Walsh shares her experience with her Whill C2 power wheelchair, which has been essential in managing her Friedreich's ataxia. Recently, her wheelchair malfunctioned due to water damage, leaving her reliant on a manual wheelchair. This change has highlighted the challenges she faces without her power chair, such as difficulty transferring into bed and rescheduling activities. Despite these setbacks, Jean remains positive, emphasising the freedom her wheelchairs provide. She rejects terms like "wheelchair-bound," preferring "wheelchair user" to describe her mobility. Her story underscores the importance of mobility aids in maintaining independence and the resilience required to adapt to unexpected challenges.

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