The National Organization for Rare Disorders (NORD) held its virtual Rare Diseases and Orphan Products Breakthrough Summit on October 18-19, 2021. Patient Worthy was able to sit in on some…
Atara Therapeutics has released data from its Phase 1 open-label extension (OLE) trial of ATA188, a treatment for progressive multiple sclerosis (MS). This allogeneic T-cell immunotherapy targets B cells and…
Previously, clinical-stage genetic medicines company Homology Medicines, Inc. ("Homology") submitted an Investigational New Drug (IND) application to the FDA for HMI-103, an investigational gene editing therapy designed to treat phenylketonuria…
The 37th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) was held last week from the 13th to the 15th. This event is the largest…
According to The Guardian, England’s National Health Service (NHS) has announced that a new therapeutic option will be available for patients with sickle cell disease (SCD). This treatment, called crizanlizumab,…
In June 2021, Redx Pharma dosed its first healthy volunteer in a Phase 1 clinical trial evaluating the safety and phamacological profile of RXC007. Altogether, this investigational therapy is designed…
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