News and Treatment Updates

Here's where you'll find a regularly updated, broad range of articles written by the AAMDSIF team, allied health organizations and news organizations. By staying well-informed, patients and families are practicing a form of self-support that will help them be more effective self-advocates when engaging with health care providers.

Science Simplified: What is a Natural History Study?

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Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science accessible and empower rare disease community members with scientific knowledge. Dr…

Monitoring Amphiregulin May Be Warranted in Caring for Patients with Acute GVHD

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Amphiregulin (AREG) may represent a useful longitudinal monitoring biomarker for patients with life-threatening acute graft-vs-host disease (GVHD), according to a sample of 2 prospective clinical trials that was presented at the 2021 ASH Annual Meeting. Specifically, patients…

Women With AML Have Worse Outcomes With Gemtuzumab Ozogamicin

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Combination gemtuzumab ozogamicin (GO) and cytarabine is no more effective than standard chemotherapy for older adults with previously untreated, standard-risk acute myeloid leukemia (AML), a phase 2/3 trial suggests. In fact, GO plus cytarabine was associated with worse event…

Aspaveli® (pegcetacoplan) approved in EU as orphan drug for treatment of PNH

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Published: Dec 15, 2021 STOCKHOLM, Dec. 15, 2021 /PRNewswire/ -- The first targeted C3 therapy approved in the EU Approval based on results from head-to-head PEGASUS phase 3 study where Aspaveli demonstrated superiority to eculizumab in improving haemoglobin levels1 Aspaveli will…

FDA Approves First Drug to Prevent Graft Versus Host Disease

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Today, the U.S. Food and Drug Administration approved Orencia (abatacept) for the prophylaxis (prevention) of acute graft versus host disease (aGVHD), a condition that occurs when donor bone marrow or stem cells attack the graft recipient, in combination with certain…

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