Showing 1241-1250 of 1319 results for "".
- FDA Grants Orphan Drug Designation to Exon 44 Skipping AOC for Duchenne Muscular Dystrophyhttps://practicalneurology.com/news/fda-grants-orphan-drug-designation-to-exon-44-skipping-aoc-for-duchenne-muscular-dystrophy/2470270/AOC 1044 (Avidity Biosciences, San Diego, CA), an antibody oligonucleotide conjugate (AOC), was granted orphan drug designation by the Food and Drug Administration (FDA) for the treatment of patients with Duchenne muscular
- Vyvgart Hytrulo Reduces Relapse Risk in Patients with Chronic Inflammatory Demyelinating Polyneuropathyhttps://practicalneurology.com/news/vyvgart-hytrulo-reduces-relapse-risk-in-patients-with-chronic-inflammatory-demyelinating-polyneuropathy/2470254/Analysis of topline data from the ADHERE (NCT04281472) clinical trial revealed that Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc; Argenx SE, Amsterdam, The Netherlands) treatment administered with the drug deli
- Full Results from LAVENDER Study Confirm Benefits of Daybue to Treat Rett Syndrome Symptomshttps://practicalneurology.com/news/full-results-from-lavender-study-confirm-benefits-of-daybue-to-treat-rett-syndrome-symptoms/2470208/According to new results of the LAVENDER study published in Nature Medicine, Daybue (trofinetide, Acadia Pharmaceuticals Inc., San Diego, CA) may be effective in treating and improving core symptoms of Rett syndrome
- FDA Grants Fast Track Designation for Antibody Oligonucleotide Conjugate to Treat Duchenne Muscular Dystrophyhttps://practicalneurology.com/news/fda-grants-fast-track-designation-for-antibody-oligonucleotide-conjugate-to-treat-duchenne-muscular-dystrophy/2470170/AOC 1044 (Avidity Biosciences, Inc, San Diego, CA), a drug in the new class of RNA therapeutics called antibody oligonucleotide conjugates (AOCs), was granted Fast Track designation by the Food and Drug Administration (FDA)
- New Therapy Approved for Rett Syndromehttps://practicalneurology.com/news/new-therapy-approved-for-rett-syndrome/2470139/The Food and Drug Administration (FDA) has approved trofinetide (Daybue; Acadia Pharmaceuticals, San Diego, CA) for the treatment of
- Pimavanserin Lowers Mortality Risk in Those with Parkinson Disease Psychosishttps://practicalneurology.com/news/pimavanserin-lowers-mortality-risk-in-those-with-parkinson-disease-psychosis/2470091/Results from a retrospective analysis published in Drug Safety indicate that individuals with Parkinson disease psychosis (PDP) treated with pimavanserin (Nuplazid; Acadia Pharmaceuticals, San Diego, CA) have a lower mortality risk compared with those treated with other atypical an
- New FDA Filing for Potential Drug To Treat Chorea Associated with Huntington Diseasehttps://practicalneurology.com/news/new-fda-filing-for-potential-drug-to-treat-chorea-associated-with-huntington-disease/2470090/The Food and Drug Administration (FDA) has accepted a supplemental New Drug Application (sNDA) for valbenazine (Ingrezza; Neurocrine Biosciences, San Diego, CA) as a treatment for chorea associated with Huntington disease (HD). The sNDA filing contained data from the KINECT-HD phase 3 study (
- Study Finds Evidence of Link Between Absence Seizures and Myelin Plasticityhttps://practicalneurology.com/news/study-finds-evidence-of-link-between-absence-seizures-and-myelin-plasticity/2470079/Results from a novel study presented at the American Epilepsy Society Annual Meeting revealed an association between seizures and myelin plasticity, opening exciting possibilities for future research. Researchers documented an increase in both the total number of myelinated fibers as well as thic
- Opicapone Evaluated for Patient Characteristics, Treatment Patterns, and Safety/Tolerability in Individuals with Parkinson Diseasehttps://practicalneurology.com/news/opicapone-evaluated-for-patient-characteristics-treatment-patterns-and-safetytolerability-in-individuals-with-parkinson-disease/2470060/In the Opicapone Treatment Initiation Open-Label Study (OPTI-ON) study, participants treated with opicapone (Ongentys; Neurocrine Biosciences, San Diego, CA) for Parkinson disease (PD) "OFF" episodes were observed for patient characteristics, treatment patterns, and safety/tolerability.
- FDA Accepts New Drug Application of Trofinetide for Rett Syndrome Treatmenthttps://practicalneurology.com/news/fda-accepts-new-drug-application-of-trofinetide-for-rett-syndrome-treatment/2470018/The Food and Drug Administration (FDA) has accepted filing of a New Drug Application (NDA) for trofinetide (Acadia Pharmaceuticals, San Diego, CA) for the treatment of Rett syndrome. Support for the application comes from the pivotal phase 3 Lavender study (NCT04181723), the results of which were