The aHUS Alliance Global Action team remains strong advocates for those affected by the rare disease atypical HUS, and proudly partners with people and groups dedicated to shedding light on clinical care, filling in knowledge gaps, and working together to build a brighter future for those affected by this rare disease.
We’re committed to tracking and sharing the latest in aHUS research and clinical updates, grounded in the fact that our international crew is all-volunteer with ‘skin in the game – we’re also aHUS families ourselves (patients and caregivers). Interestingly, and unlike our past ‘research roundups’, there’s a widespread range of topics published which are of high interest in the aHUS community.
Several dozen more new articles have been added to the ‘New Research’ scroll in our aHUS Virtual Library, with only a few are noted here. We’ve sorted over 1,500 aHUS-specific publications in 21 categories for people who wish to explore a specific topic such as transplants, pregnancy, or extra renal involvement- the impact on organs such as the heart, brain, or GI tract. (FMI on our aHUS Virtual Library,see below)
Let’s look at just a small selection of these recently released publications.
Impact on Patient & Family Life
Living with atypical HUS means managing an unpredictable and potentially life-threatening disease that can suddenly relapse, often after a long and stressful diagnostic journey. Many patients experience chronic kidney damage, fatigue, and reduced quality of life. Atypical HUS affects family life beyond the patient and caregiver roles, in areas such as attendance and focus at work or school to negative impacts on mental wellness. Aside from health concerns and related issues such as potential for increased infection vulnerability, financial stress and juggling logistics related to medical care can strain relationships and limit quality of life within the family unit and far beyond.
Limited awareness among healthcare providers given the rarity of the disease, as well as social/emotional/economic burdens on aHUS families, add to the daily challenges and make aHUS an especially difficult rare disease to navigate both medically and personally.
Palagyi A et al. Systematic Scoping Review of Socioeconomic Burden and Associated Psychosocial Impact in Patients With Rare Kidney Diseases and Their Caregivers. Kidney International Reports, Vol 10, Issue 3, 838 – 854. doi: 10.1016/j.ekir.2024.12.005
Dumbuya JS et al. The impact of rare diseases on the quality of life in paediatric patients: current status. Front Public Health. 2025 Mar 24;13:1531583. doi: 10.3389/fpubh.2025.1531583.
Biosimilars: Drug Access & Affordability
Let’s face a harsh reality. Most nations have limited financial resources for medical care, with the bulk of that dedicated to therapeutic options which serve patients with more common medical conditions than aHUS (which affects only a handful of people per million population). When complement inhibitors were approved in those nations, part of the approval process was to address whether the treatment was life-long, or whether/if under specific conditions and guidelines the drug could be discontinued.
While we’ve listed such publications on treatment and relapse risk assessments within our scroll “Discontinuing Treatment/Relapse” (ASK JEN), treatment with drugs similar to the biologic drug eculizumab has been studied. A ‘biosimilar’ is a biologic medicine derived from living cells that is highly similar—but not identical—to an approved ‘reference’ biologic drug (like eculizumab), with no clinically meaningful differences in safety, purity, or effectiveness. In resource-limited healthcare systems, biosimilars can enhance treatment equity, since the high cost of reference biologics often restricts availability.
Biosimilars like Epysqli® (eculizumab‑aagh) and Bkemv® (eculizumab‑aeeb) may help ensure aHUS and other rare disease therapies are more broadly reimbursed and accessible globally, yet they’ve faced challenges which include limited adoption. Here are two recent publications that highlight key findings and offer a generalized overview of policy implications.
Jang JH et al. The Path to Accessible Care: Development and Impact of Eculizumab Biosimilars for Paroxysmal Nocturnal Hemoglobinuria and Atypical Hemolytic Uremic Syndrome. BioDrugs. 2025 Mar;39(2):281-295. doi: 10.1007/s40259-025-00707-3.
Kvien TK et al. Beyond Cost: Observations on Clinical and Patient Benefits of Biosimilars in Real-World Settings. BioDrugs. 2025 Jul;39(4):537-553. doi: 10.1007/s40259-025-00727-z.
Note: Use our website’s Search Bar to find featured articles about aHUS and biosimilars.
Patient Advocacy: Insights that make a Difference
Patient advocates and organizations play a vital role in connecting the lived experiences of those affected by aHUS with research teams, clinicians, and industry. Seeking out and engaging with atypical HUS patients and advocacy groups ensures that research priorities, clinical trial design, and treatment development reflect real patient needs and daily challenges. For rare diseases like aHUS, where limited data and small patient populations complicate research, actively listening to patient voices helps identify unmet needs, improve disease awareness, and shape policies that expand timely access to diagnosis and treatment—ultimately leading to more patient-centered, effective, and equitable care.
We’re pleased to present a recent example of integrating aHUS patients into research, with this consensus document of recommendations from Italian scientific societies, patient associations and regulators.
Stea ED et al. Multidisciplinary consensus on the diagnosis and management of patients with atypical Hemolytic Uremic Syndrome (complement-mediated TMA): Recommendations from Italian scientific societies, patient associations and regulators. Pharmacological Research, Vol 216, 2025, 107714, ISSN 1043-6618, doi.org/10.1016/j.phrs.2025.107714.
Congratulations to all involved with this publication, with our sincere appreciation to Paolo Chiandotto and the Italian aHUS organization ‘Progetto Alice Associazione per la lotta alla SEU’ for sharing insights on behalf of aHUS patients and their families.
A ‘Virtual Library’ of aHUS-specific Research – What is that?
Often publications with advancements and insights into this very rare disease are scattered across medical journals related to nephrology and hematology specialties, but atypical HUS information can be found under varied terms and across specialty areas. That’s where we come in, since as aHUS patients and family caregivers ourselves we have a vested interest in staying up-to-date with current research. It’s likely that other families affected by aHUS may wish to key into a specific topic, so to aid navigation of our ‘Research and Publications’ page we’ve not only categorized entries but also have added links to jump to each Scroll (topic section) to speed your search.
Research articles about atypical HUS are listed within our website on topic-specific lists within our Info Centre, where you can see a list for “New Research” which are then also listed by category. Scroll topic categories are: New Research, Critical Care, Diagnosis, Treatment, Discontinuing Treatment/Relapse, Thrombotic Microangiopathy (TMA), Extra Renal (Effects on Organs other than Kidneys), Pregnancy, Transplants, Genetics, Complement, Secondary aHUS, Triggers, Research in Specific Nations, Case Studies, Patient Registries: Publications, Drug Discovery/Research, Summary Articles/Literature Reviews, Consensus Documents/Guidelines, and Varied Topics.
(Note: All entries from the ‘New Research’ scroll additionally are cross-listed at the start of each specific topic. While this ‘library’ has grown to over 1,500 unique publications, it results in more than 1800 dual entries – such as a study that examines mutation impact on disease management, and thus listed on both Genetics and Treatment scrolls.)
Visit our ‘virtual library’ of over 1,500 publications specific to aHUS
by clicking this link: Atypical HUS Research & Publications
Keeping Pace with Advancements: Are you ‘aHUS Aware’?
The aHUS Alliance Global Action team encourages researchers to release their studies and publications in full text versions, with Open Access to facilitate sharing knowledge. Additionally, we welcome and applaud those individuals and groups who include “Plain Language Versions” as part of their publication’s Supplementary Materials.
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