Elypta announces the enrollment of the first patient in the newly initiated AURORAX-0093A (AUR93A) study. AUR93A is intended to explore Elypta’s metabolism-based liquid biopsy platform for the prognosis of muscle-invasive bladder cancer (MIBC) before neoadjuvant chemotherapy. The first patient was enrolled at San Raffaele Hospital, Milan, Italy. AUR93A In AUR93A,...
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HYOGO, Japan – JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced today that European Medicines Agency (EMA) has granted orphan drug designation to JR-171, an investigational drug for the treatment of mucoplysaccharidosis type I (MPS I, or Hurler, Hurler-Scheie and Scheie syndrome). JR-171 is a blood-brain-barrier...
CASTELVECCHIO PASCOLI, Italy — Kedrion announces that the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to its investigational treatment for Congenital Aceruloplasminemia (ACP), a rare genetic disorder of iron metabolism. Aceruloplasminemia is an autosomal recessive disorder caused by mutations in the CP gene, leading to a deficiency or...
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...
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...