GoBroad (Guangdong) Rare Disease Center Launches Innovative “Full-Cycle Support” Model, Advancing Systematic Solutions for Rare Diseases

Summary: Ahead of Rare Disease Day 2026, the GoBroad (Guangdong) Rare Disease Center was officially inaugurated at Dongguan Taixin Hospital. The center brings together multidisciplinary care and precision genetic diagnostics, with hematopoietic stem cell transplantation (HSCT) as a core capability and broader treatment pathways including CAR-T and other immunotherapies. At the same event, the Guardian AnAn 2.0 charitable support program was launched, expanding assistance from basic financial support to coverage for serious complications during transplantation, second-transplant support, and humanitarian assistance. The program is designed for families affected by thalassemia, aplastic anemia, and primary immunodeficiency disorders. Prof. Chunfu LI said the model is intended to address a common challenge in rare disease care: advanced medical technology may be available, but patients still need a coordinated system that supports them throughout treatment.

Some children with severe thalassemia once depended on regular blood transfusions simply to get through each day. A child with pure red cell aplasia went through repeated consultations, unsuccessful steroid treatment, and transfusion dependence before finally receiving a hematopoietic stem cell transplant. A child with neuroblastoma traveled all the way from Russia in search of a more advanced, systematic treatment strategy.

These stories once unfolded separately across different cities and departments. On February 27, 2026, on the eve of the 19th Rare Disease Day, they found a shared point of connection: the GoBroad (Guangdong) Rare Disease Center was officially inaugurated at Dongguan Taixin Hospital.

Jointly established by the GoBroad Chunfu Institute of Hematology & Oncology and Dongguan Taixin Hospital, the center is a regional platform for rare disease diagnosis and treatment. With HSCT as a core clinical capability, it integrates multidisciplinary care and precision genetic diagnostics, expands comprehensive treatment pathways including CAR-T and other immunotherapies, and connects medical care with charitable support and long-term follow-up. The goal is to move rare disease care toward a more coordinated and systematic model. Under the theme “Advancing Systematic Solutions for Rare Diseases,” the launch event also introduced the Guardian AnAn 2.0 charitable support program and featured patient stories and expert media discussions.

The GoBroad (Guangdong) Rare Disease Center Is Officially Inaugurated

From Specialized Expertise to System Building: Rare Disease Care Enters a More Integrated Stage

Rare diseases may be individually uncommon, but the real challenge is making sure patients can access coordinated care.

More than 7,000 rare diseases have been identified worldwide, and over 80% are associated with genetic factors. In China, the number of people living with rare diseases is estimated to exceed 20 million. Because each individual condition affects relatively few people, recognition at the primary-care level may be limited and care pathways can be fragmented. As a result, many families spend years seeking a definitive diagnosis.

Hematopoietic stem cell transplantation is currently one of the most established curative treatment options for several rare hematologic disorders, including thalassemia, aplastic anemia, and primary immunodeficiency disorders, and transplant-based strategies are also used in selected malignant rare diseases such as neuroblastoma. With advances in haploidentical transplantation, the lack of a fully matched donor is no longer the barrier it once was for many families.

But advances in technology do not automatically translate into better outcomes for every patient. Even when a donor is available, families may still face financial pressure. Serious infections or other complications can arise during transplantation. Some patients may need another transplant, while others may struggle with the long-term costs of medication and follow-up after discharge. These are real barriers that can interrupt care and prevent patients from fully benefiting from available treatments.

At the inauguration ceremony, Prof. Chunfu LI, President of the GoBroad Chunfu Institute of Hematology & Oncology, said that the central purpose of establishing the rare disease center is to bring resources together through a platform-based model and address a key gap in the field: medical technology may be in place, but the surrounding care system may still be incomplete.

Prof. Chunfu LI, President of the GoBroad Chunfu Institute of Hematology & Oncology, Speaks at the Inauguration Ceremony

“Building a rare disease center is, at its core, about building a complete care system: multidisciplinary collaboration, standardized clinical pathways, regional referral networks, and long-term follow-up data. These are not just formalities; they can directly affect survival and quality of life,” Prof. LI said. The GoBroad (Guangdong) Rare Disease Center will bring together expertise in hematology, pediatrics, rheumatology and immunology, oncology, imaging, critical care, and genetic diagnostics. Through a multidisciplinary team (MDT) model, standardized transplant pathways, regional referral collaboration, and long-term follow-up, the center aims to connect diagnosis, treatment, and survivorship management into one coordinated continuum of care.

Innovating Charitable Support: Staying With Families Throughout the Transplant Journey

Another major announcement at the event was the launch of the Guardian AnAn 2.0 charitable support program.

Ms. Yong MENG, Chair of the Beijing Zhongci Public Welfare Foundation, Introduces the Guardian AnAn 2.0 Charitable Support Program

Supported by the Beijing Zhongci Public Welfare Foundation, the program upgrades the previous one-time assistance model into a full-cycle support system covering the entire HSCT journey. It is available to eligible families of patients with thalassemia, aplastic anemia (including pure red cell aplasia), and primary immunodeficiency disorders (PID) who undergo HSCT at the GoBroad Chunfu Institute of Hematology & Oncology.

The upgraded program provides four levels of assistance: basic support when patients enter the transplant pathway (RMB 10,000-30,000); additional support if serious infections, GVHD, or other complications occur during the peri-transplant period (RMB 10,000-30,000); dedicated assistance for complex cases requiring a second transplant (up to RMB 100,000); and humanitarian assistance for exceptional medical circumstances, activated after individual assessment.

The new provision of up to RMB 100,000 for a second transplant drew particular attention. “At the GoBroad Chunfu Institute of Hematology & Oncology, we want to take responsibility for every patient who chooses us,” Prof. LI said. “That responsibility is not only about pursuing better medical technology. It is also about whether we can stand with families when the treatment journey becomes more difficult, help share some of the risk, and reduce the chance that financial pressure forces a family to stop treatment. We do not want practical hardship to become the reason someone has to give up.” He added that the team has confidence in its medical and technical capabilities and expects that only a small number of families will ultimately need this higher level of follow-up assistance.

During the event, Ms. Yong MENG, Chair of the Beijing Zhongci Public Welfare Foundation, emphasized the close connection between charitable support and medical care. Stable financial assistance can help patients stay on treatment and reduce interruptions caused by overwhelming financial pressure, supporting greater continuity and quality of care. Medical advances create treatment opportunities, while charitable programs can lower the barriers to accessing and completing care. Together, they can form a more sustainable support system for rare disease patients and families.

Patient Stories: From Facing Rare Disease Alone to Receiving Systematic Support

At the inauguration ceremony, four patients and family members shared their experiences, turning medical progress into stories of everyday life, treatment, and recovery.

Xiao Zhong, 18, and Xiao Qin, 23, had spent much of their childhood and adolescence living around blood transfusions and iron chelation for severe thalassemia. As they entered adulthood, both chose hematopoietic stem cell transplantation in the hope of ending lifelong transfusion dependence. As transplantation techniques have matured and donor options have expanded, more older adolescents and adults with thalassemia may now have the opportunity to pursue curative treatment and move toward the future with greater independence.

The importance of social support beyond medical technology was also reflected in the story shared by the mother of a child with pure red cell aplasia (PRCA). Because PRCA is rare and can be difficult to diagnose, the family went through a long diagnostic journey before the child was fortunate to find a donor through the China Marrow Donor Program and successfully undergo transplantation. After recovery, the mother created a patient mutual-support group. Having once received help herself, she began helping other families by sharing experience, offering emotional support, and connecting them with useful information.

The final story came from the family of a child with neuroblastoma who traveled from Russia to China for treatment. Their cross-border journey involved close multidisciplinary collaboration between the GoBroad Chunfu Institute of Hematology & Oncology and Sun Yat-sen University Cancer Center. The expert team developed a systematic and individualized treatment plan that included immunotherapy. This collaboration across institutions and specialties illustrates how coordinated care can support patients with especially complex rare diseases.

A Russian Patient Celebrates Chinese New Year in China and Shares Good Wishes

Together, these four stories span nonmalignant and malignant disease, domestic and international patients, and children, adolescents, and young adults. They reflect a broader shift in rare disease care: from passive waiting to earlier and more active intervention; from one-time treatment to long-term survivorship and quality-of-life management; and from families feeling isolated to patients being supported by a coordinated system.

Clinical Innovation: Expanding Both Safety and Treatment Options

Experts Discuss Advances in Rare Disease Diagnosis and Treatment

During the expert discussion that followed, clinicians from several specialties shared recent developments in rare disease care.

Limited donor availability was once one of the biggest barriers preventing families from moving forward with transplantation. As haploidentical transplantation has matured, a parent can often serve as a donor, substantially reducing the time patients may otherwise spend waiting for a registry match.

More systematic peri-transplant management has also played a major role in improving transplant safety. High-resolution HLA typing, optimized GVHD-prevention strategies, standardized infection prevention and control, quantitative monitoring, and preemptive treatment have all contributed to reducing transplant-related mortality.

On the treatment side, CAR-T and other targeted immunotherapies are increasingly being integrated with transplant strategies, creating additional options for patients with relapsed or refractory disease. In high-risk neuroblastoma, multimodal approaches combining chemotherapy, targeted therapy, and transplantation are being used more widely, while CAR-T therapy for glioma remains an area of active exploration. By bringing cases and multidisciplinary expertise together, the GoBroad (Guangdong) Rare Disease Center aims to build local clinical experience and develop care pathways suited to patients in China.

Prof. Chunfu LI noted that as medical technology advances, the focus in rare disease care is shifting from whether a disease can be treated to how to treat it better and help patients live longer. This requires earlier recognition, coordinated referrals, standardized follow-up, and family-centered support - the areas the GoBroad (Guangdong) Rare Disease Center aims to address through a systematic model.