Genetic Diseases News

Alnylam Pharmaceuticals announced a new and enhanced framework for value-based agreements designed to provide access to the recently FDA-approved Givlaari for patients with acute hepatic porphyria, according to a press release.

The FDA approved Givlaari for the treatment of adults with acute hepatic porphyria, a rare genetic disorder that leads to buildup of toxic porphyrin molecules that form during the production of heme.

SAN ANTONIO — The National Liver Congress will convene October 25th, providing a broad scientific program and exhibit of products and services important for all practitioners involved in the care of patients with liver disease.

CHICAGO — Therapeutic intervention with endoscopic retrograde cholangiopancreatography in children with pancreas divisum demonstrated a high technical success rate, according to a presentation at NASPGHAN 2019.

CHICAGO — In this exclusive video from NASPGHAN 2019, Hassan M. Abdullah, MD, from the Rainbow Babies and Children’s Hospital in Cleveland, Ohio, discusses details from his poster presentation on the correlation between adenomatous polyposis coli gene mutations with pediatric hepatoblastoma.

The American College of Gastroenterology released a new clinical guideline for the screening, diagnosis and treatment of hereditary hemochromatosis, a genetic disease that alters the body’s ability to regulate iron absorption and one of the most common genetic disorders among people of northern European descent.

The American Association for the Study of Liver Diseases Foundation has granted $3.42 million in research and career development awards to 29 researchers and clinicians to conduct liver disease research and undergo advanced hepatology training.

Researchers found that excess liver iron was most likely a systemic problem related to genes rather than an organ-related problem, according to a presentation at this year’s conference of the European Society of Human Genetics.

CAMP4 Therapeutics and Alnylam Pharmaceuticals announced a collaboration designed to identify new medication targets of an undisclosed rare liver disease with significant unmet need, according to a press release.

WASHINGTON — Beginning Friday, October 20, Healio Gastroenterology and Liver Disease will provide live coverage from the The Liver Meeting 2017.