– Evrysdi is the first and only at-home administered treatment for patients living with SMA – Evrysdi has proven efficacy in babies, children and adults with more than 5,000 patients treated to date PTC Therapeutics, Inc. announced on May 31st 2022 that the US Food and Drug Administration (FDA) has approved a label extension for Evrysdi® (risdiplam) to…
NICE recommends Sleepio app for insomnia
Sleepio is recommended as a cost saving option for treating insomnia and insomnia symptoms in primary care for people who would otherwise be offered sleep hygiene or sleeping pills. For people who may be at higher risk of other sleep disorder conditions, such as in pregnancy, or in people with comorbidities, a medical assessment should…
Positive CHMP opinion for Upstaza™ for the treatment of AADC deficiency
PTC Therapeutics, Inc. announced on May 20th that Upstaza™ (eladocagene exuparvovec; PTC-AADC) received a positive opinion by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). Once ratified by the European Commission, Upstaza will be the first approved disease-modifying treatment for aromatic L-amino acid decarboxylase (AADC) deficiency for patients…
A music based walking app for MS?
Biogen is looking to utilise a music-based digital therapeutic to help improve the gait of multiple sclerosis patients who may have difficulty walking. Developed by MedRhythms, the prescription app aims to do more than simply connect the beat of the music with walking cadence. The US-based company has been working on sensors and rehabilitation software…
Treating seizures associated with Lennox-Gastaut Syndrome
JAMA Neurology publishes Phase 3 study results on the efficacy and safety of FINTEPLA®▼(fenfluramine) oral solution for the treatment of seizures associated with Lennox-Gastaut Syndrome (LGS) Primary endpoint was met demonstrating that fenfluramine, as adjunctive treatment, is effective in significantly reducing the frequency of drop seizures in LGS patients compared to placebo1 LGS is a…
NMOSD trial success
ULTOMIRIS® (ravulizumab-cwvz) Met Primary Endpoint in CHAMPION-NMOSD (Neuromyelitis Optica Spectrum Disorder) Phase III Trial Positive high-level results from the open-label Phase III CHAMPION-NMOSD trial showed that ULTOMIRIS®(ravulizumab-cwvz) achieved a statistically significant and clinically meaningful reduction in the risk of relapse in adults with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD) compared to the external placebo…
New Three-Year Data for Genentech’s Evrysdi (risdiplam)
Long-term improvements in survival and motor milestones in babies with Type 1 Spinal Muscular Atrophy (SMA) – 91% of infants treated with Evrysdi in the FIREFISH study were still alive at three years – – Infants treated with Evrysdi maintained or continued to improve in measures of motor function, including their ability to sit without…
Keto diet improved life quality of RRMS patients in trial
Eating a ketogenic diet — one low in carbohydrates and high in fats — led to less fatigue and depression for people with relapsing-remitting multiple sclerosis (RRMS) in a small clinical trial that was designed to assess the tolerability of the dietary intervention. Measures of disability and quality of life also improved during the study while participants…
NfL test for multiple sclerosis
Quanterix Granted Breakthrough Device Designation from U.S. FDA Blood-based assay has the potential to serve the multiple sclerosis (MS) community in management of relapsing-remitting form of the disease April 22, 2022 08:30 AM Eastern Daylight Time: Quanterix Corporation, a company digitising biomarker analysis with the goal of advancing the science of precision health, announced that…
