This post was originally published on this site The U.S. Food and Drug Administration (FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history — how disorders such as spinal muscle atrophy (SMA) run their course if untreated — the choice of “efficacy endpoints” in clinical…
This post was originally published on this site Deaths attributed to sarcoidosis as the primary underlying cause increased between 1999 and 2016 in the United States, according to a recent observational study. The study, “Sarcoidosis deaths in the United States: 1999–2016,” published in the journal Respiratory Medicine, compared death rates among different regions, races, genders,…
This post was originally published on this site Two cardiologists in the Netherlands are using an artificial intelligence (AI) tool called the VMS Heart Analysis System, developed by Ventripoint Diagnostics, for the early diagnosis of pulmonary hypertension in sarcoidosis patients. With this tool, Marco Post, MD, PhD, and Marloes Huitema, MD, at the St. Antonius…
This post was originally published on this site A research team at Yale School of Medicine successfully treated a person with disfiguring sarcoidosis with Xeljanz (tofacitinib) — an existing approved treatment against rheumatoid arthritis — resulting in a near-complete disappearance of skin lesions. The findings were published in the New England Journal of Medicine in…
This post was originally published on this site The National Cancer Institute (NCI) has awarded a five-year $12.5 million grant to City of Hope that may help improve treatment by using a vaccine, develop an immunotherapy to shut down a gene involved in B-Cell non-Hodgkin’s lymphoma, and understand who is at risk of developing a…
This post was originally published on this site Exposure to glyphosate, a common broad-spectrum herbicide used worldwide, increases the risk of non-Hodgkin’s lymphoma (NHL) by 41 percent in humans, a study says. The study, “Exposure to Glyphosate-Based Herbicides and Risk for Non-Hodgkin Lymphoma: A Meta-Analysis and Supporting Evidence,” were published in Mutation Research/Reviews in Mutation…
This post was originally published on this site I-Mab Biopharma is planning the first-in-human U.S. clinical study evaluating the safety, tolerability, and efficacy of its investigational candidate TJC4 as a treatment for solid tumors and lymphoma. The Phase 1/1b trial will explore TJC4 alone and in combination with other therapies, and is expected to launch…
This post was originally published on this site A Phase 1 trial testing the combination of ADCT-402 (loncastuximab tesirine) with Imfinzi (durvalumab) in multiple types of advanced non-Hodgkin’s lymphoma has dosed the first patient. The study (NCT03685344) is for adults with advanced diffuse large B-cell lymphoma (DLBCL), mantle cell lymphoma (MCL), or follicular lymphoma (FL) who have relapsed or…
This post was originally published on this site Poteligeo (mogamulizumab), an approved treatment for relapsed or refractory adult T-cell leukemia-lymphoma (ATL) patients in Japan, is also safe and effective in a real-world clinical setting, a postmarketing surveillance study found. The study, “Safety and effectiveness of mogamulizumab in relapsed or refractory adult T-cell leukemia-lymphoma,” was published in…
This post was originally published on this site U.S. regulators have granted orphan drug status to TP Therapeutics’ TPX-0005 as a treatment for non-small cell lung cancers with certain gene mutations, according to a company press release. The Federation Drug Administration decision is aimed at accelerating TPX-0005’s approval. The designation will also give the company seven years of…
