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Early-onset LAMA2-related muscular dystrophy occurs in approximately 1 in 30,000 individuals and accounts for between 30 and 40 percent of total cases of congenital muscular dystrophy. TXA127 is

bb2121 is a chimeric antigen receptor T cell (CAR T) therapy targeting B cell maturation antigen (BCMA), and bluebird bio is developing bb2121 in collaboration with Celgene Corporation.

The agency’s letter stated that the company’s application was not sufficiently complete and it is seeking additional supporting information. There was no comment on the acceptability of the

The designation, which is meant to expedite the development of therapies that provide benefits over existing options, was based on an early clinical data from a phase I

Inovio is developing its Zika vaccine with GeneOne Life Sciences and academic collaborators. In the pre-clinical study, Inovio’s SynCon vaccine technology was used to synthetically generate DNA vaccine

Carmot will continue to apply its proprietary lead-identification technology, Chemotype Evolution, to further advance molecules discovered during the initial collaboration. Amgen will be solely responsible for the clinical

The randomized, controlled, Phase 3 study (Protocol NCT01696084), conducted at 39 centers in the United States and Canada, compared VYXEOS to the conventional cytarabine and daunorubicin treatment regimen

Blood banks in the US have been urged to reject donations from people who have traveled to areas where the Zika virus is active or from those who

The endpoint was met at week 12 against placebo achieved p value of <0.0001. Zilretta also demonstrated significant analgesia against placebo at weeks 1 through 16. Flexion said