Videos

An expert discusses the importance of collaborating with patients, emphasizing the value of leading with data when educating them on the efficacy of treatments, and highlights the role of providers in supporting patients as they discover the medications and supplements that work best for them, while advocating for and protecting patients to ensure they can seek the best quality of life.

1 expert in this video

An expert discusses how omaveloxolone is a nuclear factor erythroid 2-related factor 2 (Nrf2) activator that addresses oxidative stress and mitochondrial dysfunction in Friedreich ataxia. It works by binding to Keap1, which releases Nrf2 to enter the nucleus and upregulate antioxidant genes. Clinical trials showed modest improvements in neurological function, as measured by modified Friedreich Ataxia Rating Scale scores. Adverse effects include headache, nausea, and elevated liver enzymes. Omaveloxolone has been integrated into clinical practice as part of multidisciplinary care, requiring regular monitoring and coordination with physical therapy, cardiology, and other specialties.

Panelist discusses how managing bladder and sexual dysfunction in neurological care requires addressing access barriers, fostering open communication, recognizing symptom overlap, understanding medication adverse effects, and providing gender-specific care.

An expert discusses upcoming advancements in Parkinson disease treatments, including the potential release of the ND0612 pump later this year, demonstrating efficacy comparable to oral carbidopa/levodopa, the dopamine agonist tavapadon, CVN424 as a novel therapy not targeting the dopamine pathway, and ongoing stem cell and gene therapy developments, with stem cells capable of creating dopamine-producing cells entering phase 3 studies.

Panelist discusses how a neurologist typically manages neurogenic bladder when it stems directly from neurological conditions, they’re already treating but that they should refer to urology for complex cases requiring specialized urologic interventions, when conservative management fails, or when there are complications such as recurrent urinary tract infections or structural changes. Close collaboration between both specialists often provides optimal patient care.

1 expert in this video

An expert discusses how Friedreich ataxia significantly impacts quality of life through progressive neurological deterioration affecting mobility, coordination, and cardiovascular health. Standard care includes symptom management with physical/occupational therapy and cardiac monitoring for both pediatric and adult patients. Multidisciplinary teams (neurologists, cardiologists, therapists, and genetic counselors) coordinate comprehensive care addressing the disease's multisystem effects.

1 expert in this video

An expert discusses how Friedreich ataxia is an autosomal recessive neurodegenerative disorder caused by trinucleotide (GAA) repeat expansion in the FXN gene, reducing frataxin protein production. This leads to mitochondrial dysfunction and progressive damage to the spinal cord, peripheral nerves, and heart. Patients present with progressive ataxia, dysarthria, sensory loss, and areflexia, typically manifesting between ages 5 and 15. Diagnosis relies on clinical assessment, genetic testing for GAA expansions, and supportive findings, including cardiomyopathy, scoliosis, and diabetes.

An expert discusses the extended form of carbidopa/levodopa (Crexont) for managing OFF episodes, highlighting trial results that show decreased OFF time and increased ON time, as well as the use of subcutaneous loading of medications in Parkinson disease, which was well tolerated with skin issues as the main adverse effects and resulted in increased ON time without worsening dyskinesia.