NEW YORK, NY – Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted its allogeneic cell therapy Revascor® (rexlemestrocel-L) an Orphan-Drug Designation (ODD) following submission of results from the randomized controlled trial in children...
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Copenhagen, Denmark – Zealand Pharma A/S, a biotechnology company focused on the discovery and development of innovative peptide-based medicines, today announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for Part 1 of the New Drug Application (NDA) for dasiglucagon for the prevention...
It’s shortly after 5 a.m. when the phone rings, and on the line is a clearly anxious and worried parent. “Sierra is having a lot of problems tonight,” Shaylene Akery tells a CNN producer. “We have to take her to the hospital, but we still want to talk to you...
United States Sens. Tim Kaine (D-VA), Mark R. Warner (D-VA), Jerry Moran (R-KS) and Bill Cassidy (R-LA) introduced Thursday the Gabriella Miller Kids First Research Act 2.0, which would provide additional funding for childhood cancer research. The bill is named in honor of Gabriella Miller, a Leesburg resident who died...
BIRMINGHAM, Ala. – The University of Alabama at Birmingham startup TIXiMED, Inc., has obtained clearance from the U.S. Food and Drug Administration to proceed to clinical trials under an Investigational New Drug for TIX100, its novel oral Type 1 diabetes drug. This represents a major milestone in the development of...
Davis, California – A recent study led by UC Davis Health researchers provides new insights into the molecular changes linked to the rare genetic condition 22q11.2 deletion syndrome, or 22q. It found unique biomarkers that could identify patients with 22q who may be more likely to develop schizophrenia or psychiatric conditions, including...
RIVERSIDE, Calif. — NeuroHIV refers to the effects of HIV infection on the brain or central nervous system and, to some extent, the spinal cord and peripheral nervous system. A collection of diseases, including neuropathy and dementia, neuroHIV can cause problems with memory and thinking and compromise our ability to...
RIVERSIDE, Calif. — Nonsense-mediated RNA decay, or NMD, is an evolutionarily conserved molecular mechanism in which potentially defective messenger RNAs, or mRNAs (genetic material that instructs the body on how to make proteins), are degraded. Disruption of the NMD pathway can lead to neurological disorders, immune diseases, cancers, and other pathologies....
San Diego, Calif. – UC San Diego Health is the first health system in San Diego County to offer a new bladder-saving gene therapy to treat localized bladder cancer. The novel treatment is the first and only FDA-approved gene therapy delivered directly into the bladder for non-muscle-invasive bladder cancer (NMIBC)....
San Francisco, Calif. – In a discovery that opens the door to a less invasive way of treating some serious disorders before birth, UC San Francisco scientists have found that delivering medicine through amniotic fluid is as effective as delivering it to the fetal brain via cerebrospinal fluid. The experiment...