FDA OKs First Gene Therapy for Hemophilia B
The FDA on Tuesday approved the first gene therapy for treating hemophilia B, a genetic bleeding disorder resulting from missing or insufficient levels of factor IX. Etranacogene dezaparvovec (Hemgenix) is indicated for adults with the condition who currently use factor IX prophylaxis therapy for blood clotting, those who have or have had life-threatening hemorrhage, or […]
Biologic Shows Promise in Ongoing Food Allergy Trial
LOUISVILLE, Ky. — Omalizumab (Xolair) may be an effective therapy for patients with multiple food allergies, according to an ongoing phase III trial. Most of the first 60 patients with multiple food allergies enrolled in stage one of the OUtMATCH clinical trial were able to tolerate serving-sized portions of those foods a year after receiving […]
Promising Results in First Test of In Utero Enzyme-Replacement Therapy
The first test of in utero enzyme replacement therapy (ERT) proved safe and effective for a baby with infantile-onset Pompe disease, according to a case report. An infant with cross-reactive immunologic material (CRIM)-negative infantile-onset Pompe disease — the most severe end of the disease spectrum — had no cardiac manifestations following treatment, compared to her […]
So, You Just Had A Breakthrough In Therapy. Now What?
For most people, therapy is a time that’s dedicated to whatever the patient wants, whether that’s venting about a loved one, talking about a stressor at work or diving deep into monumental events in your childhood. Oftentimes, what comes from these deep conversations are uncomfortable truths about yourself — how your behavior contributes to a […]
Study Refutes Heart Health Claims of Dietary Supplements
CHICAGO — For people without heart disease, preventive low-dose statin therapy lowered LDL cholesterol to a degree that dietary supplements and placebo couldn’t match, a randomized trial found. People taking rosuvastatin 5 mg per day for 4 weeks had an average 37.9% reduction in LDL cholesterol, the only group to do better than the placebo […]
Testosterone Tied to Sustained Weight Loss in Men With Hypogonadism and Obesity
SAN DIEGO — Long-term testosterone therapy was associated with sustained reductions in weight for men with hypogonadism and class III obesity, a researcher reported. In registry data on 76 patients, those who opted to receive testosterone undecanoate experienced a 26% reduction in their body mass index (BMI) over close to a decade of follow-up, reported […]
Good, and Safe, Results Tied to Oral Testosterone in Men With Deficiency
MIAMI — Oral testosterone replacement therapy (TRT) seemed safe and effective in men with testosterone deficiency, according to a meta-analysis. Among nine studies that looked at changes in serum total testosterone, only men with testosterone deficiency who took an oral agent — predominantly testosterone undecanoate — saw a significant increase in total testosterone, with a […]
Study Suggests Potential to Skip Surgery for Selected Breast Cancer Patients
Women who chose not to have surgery after a pathologic complete response (pCR) to neoadjuvant therapy for early HER2-positive or triple-negative breast cancer (TNBC) had no recurrences during the first 2 years of follow-up, a small prospective study showed. Overall, 31 of 50 (62%) women had no evidence of residual disease by image-guided vacuum-assisted core […]
Start HIV Antiretroviral Therapy ASAP, Experts Urge
WASHINGTON — Early initiation of antiretroviral therapy (ART) in patients with HIV can lead to better outcomes, especially for those ages 35 and younger, a follow-up analysis of the START trial showed. For patients who had deferred ART until their CD4 counts were below 350 cells/mm3, there was a 21% higher risk of death or […]
Shorter Course of Radiation Therapy Proves Mettle in High-Risk Prostate Cancer
SAN ANTONIO — A shortened radiation therapy (RT) protocol for high-risk prostate cancer led to similar outcomes with no increase in toxicity compared with the standard treatment schedule, a randomized trial showed. Overall survival (OS) at 7 years was 82%, with no difference between standard RT and hypofractionation that reduced treatment duration from 8 to […]