Author Archives: Inês Martins PhD

SIDEROS Phase 3 Trial of Puldysa in Duchenne Patients Completes Enrollment

The SIDEROS Phase 3 clinical trial, investigating if oral Puldysa (idebenone) can be added to a stable glucocorticoid regimen to more effectively slow lung function decline in boys and men with Duchenne muscular dystrophy (DMD), has completed enrollment, Santhera Pharmaceuticals announced. A planned analysis demonstrated the study, while not enrolled up to the…

Pfizer Acquires Bamboo Therapeutics, Readies Clinical Trial of Gene Therapy for DMD

Bamboo Therapeutics has been fully acquired by Pfizer, considerably expanding that company’s holdings in gene therapy. With the acquisition, Pfizer gains several potential therapeutic agents for rare diseases, including one for Duchenne muscular dystrophy (DMD), soon to enter clinical testing; and one Phase 1/2 clinical stage agent, a potential best-in-class recombinant Adeno-Associated Virus (rAAV)…

Defective RNA Processing Linked to Cardiac Problems Seen in Myotonic Dystrophy Patients

Researchers have discovered the molecular mechanisms responsible for the heart dysfunctions that mark myotonic dystrophy, and reported they appear to be linked to a defective processing of the cardiac sodium channel (SCN5A) RNA. Their study, published in Nature Communications, is titled “Splicing misregulation of SCN5A contributes to cardiac-conduction delay and…