FOSTER CITY, Calif. — Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that data from the Phase 3 MARCH-PFIC study evaluating LIVMARLI® (maralixibat) oral solution in patients with PFIC was published in The Lancet Gastroenterology and Hepatology. The MARCH-PFIC study was the largest, randomized, double-blind, placebo-controlled study in patients with PFIC,...
treatment News
SOUTH SAN FRANCISCO, Calif. — Mission Bio, a leader in single-cell multi-omics solutions for precision medicine, announced a new publication led by Hervé Avet-Loiseau, MD, PhD, revealing new insights into disease progression of multiple myeloma (MM). Using Mission Bio’s single-cell DNA sequencing Tapestri® Platform, the study shows that solely targeting...
JERSEY CITY, N.J. – Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced the presentation of an analysis of the timing of disease progression milestones among commercially insured amyotrophic lateral sclerosis (ALS) patients treated with intravenous (IV) RADICAVA® (edaravone). The findings are being presented during the 2021 Virtual American Academy of Neurology...
JERSEY CITY, N.J. — Mitsubishi Tanabe Pharma Corporation (MTPA) today announced that the U.S. Food and Drug Administration (FDA) has recognized seven years of Orphan Drug Exclusivity (ODE) for RADICAVA ORS® (edaravone) based on their assessment that the oral form of edaravone constitutes a major contribution to patient care for people...
TORONTO – Mitsubishi Tanabe Pharma Canada, Inc. (MTP-CA), a subsidiary of Mitsubishi Tanabe Pharma America, Inc. (MTPA), announced today that RADICAVA® (edaravone), an infusion treatment for amyotrophic lateral sclerosis (ALS), is now reimbursed through Prince Edward Island (PEI) Pharmacare under Special Authorization. “We want to thank the Government of Prince Edward Island for their leadership in making this treatment available to patients in...
Singapore – A group of researchers, led by Nanyang Technological University, Singapore (NTU Singapore), has developed a mobile application to educate individuals living with diabetes and their caregivers with knowledge of the disease and assist them in monitoring their day-to-day activities, which includes medication, physical activity, and their diet. The...
TOKYO, Japan & WALTHAM, Mass. — Modalis Therapeutics Corporation (Tokyo Stock Exchange: 4883), a pioneering company developing innovative drugs for the treatment of rare genetic diseases, utilizing its proprietary CRISPR-based epigenome editing technology CRISPR-GNDM®, posted the preprint paper titled “Efficient and durable gene activation by Cas9-mediated epigenome editing in vivo”...
Memphis, TN – Immunotherapy using modified chimeric antigen receptor (CAR) T cells has greatly improved survival rates for pediatric patients with relapsed and recurrent leukemia. However, these therapies are not as effective in treating solid tumors and can have significant toxicity. Findings from St. Jude Children’s Research Hospital showed that...
MEMPHIS, Tenn. – Today, St. Jude Children’s Research Hospital scientists published their work on SJ3149, a compound with broad activity against many cancer types, particularly acute myeloid leukemia (AML). SJ3149 sticks to the cancer-related protein casein kinase 1 alpha (CK1α), leading to its destruction. The findings were reported in Nature...
HOUSTON — Moleculin Biotech, Inc, (Nasdaq: MBRX) (Moleculin or the Company), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat tumors and viruses, today provided a preliminary update on recent clinical activity and expected near term milestones across its clinical development pipeline in its quarterly...