Blacksburg, VA – Every 65 seconds, someone in the United States develops Alzheimer’s disease, a devastating form of dementia that affects 6.2 million Americans. Though it was initially identified almost 120 years ago, Alzheimer’s disease is a progressive neurological disorder with no cure and few treatments. It starts out with minor...
Latest News
ROCKLAND, Mass. — EMD Serono, the Healthcare business of Merck KGaA, Darmstadt, Germany, in the U.S. and Canada, today announced the presentation of two new post-hoc analyses of tertiary endpoints from the MAGNIFY-MS study, a Phase IV, prospective, open-label study evaluating the efficacy and safety of MAVENCLAD® (cladribine) tablets in...
Vancouver, Canada — The global orphan drug market size was USD 154.20 Billion in 2022 and is expected to register a rapid revenue CAGR of 12.3 % during the forecast period. Rising frequency of uncommon illnesses and increasing number of governments providing regulatory incentives for development of orphan drugs are...
LAS VEGAS — Muscular dystrophies encompass a range of muscle disorders resulting from genetic mutations. Over time, these conditions lead to muscle weakness, which hinders everyday activities. Muscular dystrophy takes various forms, each targeting specific muscle groups and manifesting at different ages with varying degrees of severity. It can either...
TORRANCE, Calif. — Emmaus Life Sciences, Inc. (OTCQX: EMMA), a leader in sickle cell disease (SCD) treatment, today announced that it has received marketing authorization from the Puerto Rico Department of Health for Endari® (L-glutamine oral powder). This approval marks a significant milestone in Emmaus’ mission to improve the lives...
ROCKVILLE, Md. – Emmes, a global, full-service Clinical Research Organization (CRO) dedicated to supporting the advancement of public health and biopharmaceutical innovation, today announced a partnership with MedGenome aimed at accelerating breakthrough treatments, powered by human genomics, for rare disease patients. “This is an exciting opportunity to partner with the...
Bath, England – When you’re an autistic teenager living with chronic pain, getting treatment for your pain can be a challenging experience. That’s according to a group of young people who’ve spoken to Dr. Abbie Jordan of the Department of Psychology and Centre for Pain Research at The University of...
Adding enasidenib to azacitidine significantly improved responses in older patients with newly diagnosed, IDH2-mutant acute myeloid leukemia (AML), according to research published in The Lancet Oncology. In a phase 1b/2 trial (ClinicalTrials.gov Identifier: NCT02677922), researchers compared enasidenib plus azacitidine with azacitidine monotherapy. The trial enrolled 107 patients with newly diagnosed, IDH2-mutant AML who were...
SOUTH SAN FRANCISCO, Calif. — Encoded Therapeutics Inc., a biotechnology company focused on developing genetic medicines for severe central nervous system (CNS) disorders, today announced the approval of its Clinical Trial Authorisation (CTA) application by the United Kingdom (UK) Medicines and Healthcare products Regulatory Agency (MHRA) for the EXPEDITION Study of...
SOUTH SAN FRANCISCO, Calif. — Encoded Therapeutics Inc., a biotechnology company focused on developing genetic medicines for severe central nervous system (CNS) disorders, today outlined the global development strategy for its lead gene therapy candidate, ETX101, for the treatment of SCN1A+ Dravet syndrome. Individuals with Dravet syndrome, the most common...