Interim results of the INSPIRE trial showed improved limb function, mobility, pain, and fatigue in patients with SORD deficiency and sustained sorbitol reduction vs placebo.
At the 12-month point of the 24-month prospective trial, patients randomly assigned to receive daily govorestat 20 mg/kg had statistically significant improvements on theFor the primary pharmacodynamic/biomarker endpoint, treated patients also had a sustained reduction in sorbitol levels that was statistically significant vs placebo.
There are currently no approved therapies for SORD deficiency, but the INSPIRE trial is testing govorestat, an aldose reductase inhibitor that prevents the conversion of glucose to sorbitol. "We found it interesting that these are proximal rather than distal components, suggesting — and this is not definitive —perhaps that there's a distal predominance in benefits," Shy said.
The company plans to discuss a potential new drug application submission with the US Food and Drug Administration based on this clinical data, a press statement noted.
Muscular Atrophies Charcot Marie Tooth CMT Charcot-Marie-Tooth Disease Hereditary Motor And Sensory Neuropathy Hmsn Peroneal Muscular Atrophy Pain Management Limbs Pain In Limb Limb Pain Mobility Neuropathy Diabetic Neuropathy Diabetic Neuropathies Diabetic Nerve Damage Patient Safety Nerve Cell Fatigue Pain Adverse Effects Side Effects Iowa Nerves Virginia
United States Latest News, United States Headlines
Similar News:You can also read news stories similar to this one that we have collected from other news sources.
Clinical Trial Of Ketamine Tablets Shows Promising Results For Treatment-Resistant DepressionAnuradha Varanasi is a freelance science writer. She writes on the intersection of health/medicine, racial disparities, and climate change. She earned an MA in Science Journalism from Columbia University in New York City.
Read more »
Eli Lilly's treatment for obesity and diabetes looks promising for another severe conditionThe Investing Club holds its 'Morning Meeting' every weekday at 10:20 a.m. ET.
Read more »
Oral Extended-Release Ketamine Promising for Treatment-Resistant DepressionExtended-release ketamine tablets taken twice weekly was effective for treatment-resistant depression with fewer side effects compared with intravenous or intranasal ketamine in a phase 2 trial.
Read more »
New home-administered treatment for binge eating disorder shows promising resultsResearchers have investigated the feasibility of a new home-administered treatment for binge eating disorder. The new treatment combines a gentle brain stimulation technique called transcranial direct current stimulation (tDCS) with a training program that targets unhelpful patterns of attention around food.
Read more »
Investigational Nasal Powder Rapidly, Effectively Cuts Migraine PainA single dose of a dihydroergotamine mesylate nasal powder formulation appears to quickly and significantly reduce migraine pain, results of a new phase 3 study showed.
Read more »
Study determining whether investigational drug can aid those with spinal cord injuries underwayScientists at the Shirley Ryan AbiilityLab in Chicago are studying whether an investigational drug can help the 300,000 Americans living with spinal cord…
Read more »