Option Care Health Inc., the largest U.S. independent provider of home and alternate site infusion services, was selected to participate in the limited distribution network of VILTEPSO (viltolarsen) for patients with Duchenne Muscular Dystrophy who are amenable to exon 53 skipping therapy.
COVID-19 tied to muscle-weakening disease; vaping risk documented
Annals of Internal Medicine, Brain, Coronavirus Disease 2019 (COVID-19), Coronavirus Infections, Coronavirus Vaccines, Doctors, E-Cigarettes, Illnesses, Immune Response, Journal of Adolescent Health, Masks, Medical Equipment, Muscle Wasting Disorders, Myasthenia Gravis (MG), Nervous System, Neuromuscular diseases, Researchers, Science, Scientific studies, VapingA roundup of some of the latest scientific studies on the novel coronavirus and efforts to find treatments and vaccines for Covid-19, the illness caused by the virus.
Argenx announced positive topline results from the company’s Phase III ADAPT clinical trial of efgartigimod for myasthenia gravis.
Biogen and Sangamo Therapeutics announced a broad global licensing collaboration deal to develop and commercialize several compounds for a range of neurological and neuromuscular diseases.
Roche Holding AG made the company’s second major gene therapy deal in a year, spending $1.15 billion to obtain the rights to Sarepta Therapeutics Inc.’s investigational drug to treat duchenne muscular dystrophy (DMD) outside the United States.
Reata Pharmaceuticals Inc.’s Part 2 portion of the MOXIe Phase 2 trial of omaveloxolone in patients with Friedreich’s ataxia (FA) met the primary endpoint of change in the modified Friedreich’s Ataxia Rating Scale relative to placebo after 48 weeks of treatment.
Rockville, Maryland-based RegenxBio entered a license deal with Pfizer for a gene therapy for Friedreich’s ataxia, the most common hereditary ataxia.
FDA Greenlights Soliris for Rare Autoimmune Disease
Anti-aquaporin-4 (AQP4) antibody, Approvals, Biomarkers, Central Nervous System, European Medicines Agency (EMA), FDA, FDA/Regulatory, Generalized Myasthenia Gravis (gMG), Hemolytic Uremic Syndrome, Immune System, Japanese Ministry of Health, Neuromyelitis Optica Spectrum Disorder (NMOSD), New Indications, Paralysis, Paroxysmal Nocturnal Hemoglobinuria (PNH), Vision problemsThe U.S. FDA gave Alexion Pharmaceuticals a thumbs-up for Soliris (eculizumab) to treat neuromyelitis optica spectrum disorder in adults that express a specific biomarker.
Top 10 Pipelines To Watch: 2019 Annual Report
Analysts, Ankylosing Spondylitis (AS), Annual Reports, Antiretroviral Drugs, Atopic Dermatitis (Eczema), Autoimmune Diseases, B Cells, Big Pharma, Biologics, Biopharma, Biotechnology, Blockbusters, BRCA Gene, BRCA mutation, Breakthrough Therapy Designation, Business, Cancer, CAR-T Therapy, Cells, Chemotherapy, Clinical Data, Clinical Trials, Collaborations, Crohn's Disease, Cystic Fibrosis, Drug Discovery, EU, European Medicines Agency (EMA), Fast Track Designation, FDA, FDA/Regulatory, February 2019, Future Blockbusters, Gene Editing, Generalized Myasthenia Gravis (gMG), Genomics, HIV, Human Genome, Immune Cells, Immune System, Immune-Mediated Diseases, Immunotherapy, Inflammatory Diseases, Injectables, Innovation, Integrase strand transfer inhibitors, Issue Archives, Japan, M&A, Med Ad News, Monoclonal Antibodies, Multiple myeloma, Neoantigens, Neurology, Neuromyelitis Optica Spectrum Disorder (NMOSD), New Drug Applications, Non-Nucleoside Reverse Transcriptase Inhibitor (NNRTI), Non-Small Cell Lung Cancer (NSCLC), Nonalcoholic Steatohepatitis (NASH), Nuclear Medicine, Oncology, Orphan Drug Designation, Paroxysmal Nocturnal Hemoglobinuria (PNH), Precision Medicine, Prescription Drug User Fee Act (PDUFA), Priority Medicines (PRIME) Designation, Priority Review Voucher, Product Pipelines, Proteins, Psoriatic Arthritis, R&D, Radioligands, Rare Disorders, Research, Rheumatoid Arthritis, RNA Interference (RNAi), Sickle Cell Disease, Small Molecules, Special Reports, T-Cells, Top 10 Pipelines, Top 10 Pipelines To Watch, Tumors, Ulcerative Colitis, Ultra Rare DiseasesThe return on R&D investment for leading biopharmaceutical manufacturers fell to a nine-year low while the U.S. FDA approved a record-breaking amount of novel medicines during 2018.
Wave Life Sciences and Takeda Form Global Strategic Collaboration to Advance Therapies for Central Nervous System Disorders
Alzheimer’s Disease, Amyotrophic Lateral Sclerosis (ALS), Biotechnology, Central Nervous System, CNS Disorders, Collaboration, Duchenne Muscular Dystrophy (DMD), Frontotemporal Dementia (FTD), Genes, Huntingtin (HTT) Gene, Huntington’s disease, Issue Archives, Parkinson’s disease, Spinocerebellar Ataxia Type 3 (SCA3)Wave Life Sciences Ltd. announced the formation of a global strategic collaboration with Takeda Pharmaceutical Company Ltd. to discover, develop and commercialize nucleic acid therapies for disorders of the central nervous system.