Sarepta Announces Positive Results for Elevidys, Gene Therapy for Duchenne
February 12th 2025Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is priced at $3.2 million per dose, produced $821 million in net product revenue for the company in 2024.
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Researchers to Clinicians: Don’t Neglect the Neuropsychiatric Symptoms of Friedreich’s Ataxia
November 27th 2024Poor balance and coordination are prominent features of Friedreich’s ataxia (FRDA). Czech researchers found that neuropsychiatric symptoms are also common among patients with FRDA.
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Wearable Devices Help Assess Friedreich's Ataxia Severity Better Than In-Clinic Assessments
November 4th 2024The ability to gather continuous, objective data on physical activity and upper limb movements may allow for a more comprehensive understanding of the daily functional capacities of patients with Friedereich's ataxia.
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Positive Pipeline Updates for Friedreich’s Ataxia
October 24th 2024After experiencing setbacks, PTC reported positive results for vatiquinone, its selective inhibitor of 15-Lipoxygenase (15-LO) enzyme, regulator of the energetic and oxidative stress pathways that are disrupted in Friedreich ataxia
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How Do Skeletal Muscle Relaxants Fit Into Pain Management?
October 7th 2024A review of skeletal muscle relaxants shows that they don't work for pain from osteoarthritis, neuropathic pain and other conditions. Some see an opportunity for more tailored therapeutic strategies, improving patient outcomes.
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Early Genetic Testing Avoids Treatment Delays for Patients with Friedreich's Ataxia
September 18th 2024Genetic and biochemical testing are crucial for improving outcomes for patients with Friedreich ataxia, according to speakers at a symposium at the European Association of Neurology Congress.
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Improving Pain Self-Management Among Rural, Older Cancer Patients
August 13th 2024The management of pain in older adults with cancer remains a significant public health concern and an unmet medical need, particularly in rural areas. This demographic often encounters specific challenges, such as limited access to healthcare services, hesitancy to talk about pain, and misunderstandings about pain management.
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