today announced that the company will present preclinical data from the PBGENE-DMD program for the treatment of Duchenne ...

clinical study of ENTR-601-44 for the potential treatment of Duchenne muscular dystrophy (DMD) in adult patients with a confirmed mutation in the DMD gene amenable to exon 44 skipping. The global ...

Capricor Therapeutics filed a BLA for deramiocel, showing promising results in slowing cardiac dysfunction in DMD ...

Imagine a cancer treatment that precisely targets malignant cells, leaving healthy ones untouched. Consider, also, a cancer treatment that corrects abnormal protein synthesis to produce healthy ...

I'm fighting a fatal disease that took the life of my brother. I work in biotech and know there are medical advances that ...

Pfizer’s decision to halt further marketing of Beqvez is further sign of the sparse patient interest in gene therapies for the bleeding condition.

At UMass Chan Medical School, researchers, doctors, patients and biomedical executives voiced their concerns over cuts to NIH ...

Pfizer is pulling further away from the gene therapy field with its decision to discontinue hemophilia product Beqvez. | ...

Wheelchairs 4 Kids, a nonprofit organization dedicated to improving the lives of children with mobility challenges, is ...

Families and doctors spoke out against the cuts at a roundtable at the UMass Chan Medical School in Worcester, Massachusetts.

The company, now based in North Beach, has lost more than $2 billion over 29 years. Its only approved drug, called roxadustat ...