Summaries of Must-Read Clinical Literature, Guidelines, and FDA Actions

SB-525 Gene Therapy Promising for Hemophilia A

Publish date:: April 17, 2019

Key clinical point: Interim data suggest that SB-525, an investigational gene therapy, may be safe and effective for patients with severe hemophilia A.

Major finding: Two of eight patients treated with SB-525 have achieved normal factor VIII levels.

Study details: A phase 1/2 trial with data on eight patients with severe hemophilia A.

Disclosures: The trial is sponsored by Sangamo Therapeutics in collaboration with Pfizer.

This Week's Must Reads

Younger adults present with more advanced esophageal adenocarcinoma,

PDAC: Tumor reduction after neoadjuvant therapy may predict postsurgical survival,

Mortality risks rise with age, infections, but not inhibitor status in persons with non-severe hemophilia A,

‘Impressive’ local control with MRI-guided brachytherapy in cervical cancer,

U.S. mothers underestimate role breastfeeding plays in curbing breast cancer,

Must Reads in Bleeding Disorders

Mortality risks rise with age, infections, but not inhibitor status in persons with non-severe hemophilia A,

Hemochromatosis variants may confer 10-fold higher risk of liver cancer,

Hemophilia, von Willebrand disease do not increase postop complications for ACL reconstruction,

Mouth splints decrease risk of post–dental extraction bleeding in hemophilia,

Low VWF levels or blood group O not linked to intracerebral hemorrhage risk,