NANNING, Feb. 28 (Xinhua) -- For seven-year-old Candy (pseudonym), the journey from her home in Vietnam to Nanning, the capital city of south China's Guangxi Zhuang Autonomous Region, which borders Vietnam, was a race against the clock.
Born with severe thalassemia, a debilitating hereditary blood disorder, the better part of Candy's life was once measured by three-week intervals of painful blood transfusions on a hospital bed and defined by the diameter of a needle and the slow drip of an IV bag.
Today, her world has opened up beyond recognition. She became one of six Vietnamese children recently discharged from the hematology department at the First Affiliated Hospital of Guangxi Medical University, following successful hematopoietic stem cell transplants, a procedure that effectively "reboots" the patient's blood-building system with healthy cells.
Her recovery is considered yet another milestone in a growing cross-border medical initiative between China and the Association of Southeast Asian Nations (ASEAN), transforming Guangxi from a high-incidence "hotspot" for rare diseases into a regional center of life-saving solutions.
The success of these pediatric cases is built on decades of experiments and clinical practices borne out of local necessity. In Guangxi, thalassemia is a significant public health challenge, with the carrier frequency averaging over 20 percent in the population.
The high prevalence has compelled local medical institutions to dedicate expertise and funding in the search for a solution at a scale few other places in the country could match, according to Zheng Baoshi, president of the First Affiliated Hospital of Guangxi Medical University.
At the heart of the efforts is the "Guangxi Scheme," a standardized transplant protocol developed by the hospital and forged in the decades-long battle against the debilitating disease.
According to hospital data, the scheme has achieved a 95 percent cure rate for severe thalassemia, a figure that stands on par with international standards.
The institution now performs the largest number of hematopoietic stem cell transplants in Guangxi, curing over 200 patients annually. These cases range from metabolic disorders like pyruvate kinase deficiency (PKD) to complex immune deficiencies.
This Saturday, landmarks across the world are lit in blue, pink, green, and purple, forming a "chain of light" to mark the 19th Rare Disease Day under the theme "More Than You Can Imagine," the day set up to shine a light on around 350 million people worldwide living with a rare condition.
While the World Health Organization (WHO) defines "rare" as affecting between 0.065 percent and 0.1 percent of the population, the sheer scale of the collective impact is staggering.
In China, where 207 rare conditions are listed, an estimated 20 million people live with rare diseases. For these patients, the "rarity" of their condition often translates into a woeful lack of recourse, a years-long odyssey to secure the right specialists who can recognize their symptoms and administer medical procedures they can afford, noted Zheng.
Seeking to move beyond disease management and toward permanent cures, Guangxi is aggressively recruiting global scientific talent. In late 2025, the hospital successfully brought Thomas Mikael Oliveberg, a prominent biophysicist from the Royal Swedish Academy of Sciences, into the fold.
A leading authority on protein folding and protein disease, Professor Mikael Oliveberg studies the biological "glitches" behind the neurodegenerative conditions like amyotrophic lateral sclerosis (ALS), a rare condition that was brought to global attention by the "Ice Bucket Challenge," a viral social media campaign.
Under the terms of the newly signed agreement with the Guangxi hospital, Professor Mikael Oliveberg will conduct on-site research at the institution for three months every year.
"Significant breakthroughs have been achieved in thalassemia diagnosis and treatment research, while steady progress has been made in promoting prevention and control models," said Zheng.
He is convinced that the institution has established a mature thalassemia diagnostic and therapeutic system, providing a full spectrum of services from screening and diagnosis to transplantation. Furthermore, the hospital has pioneered the internationally original "Guangxi Scheme," offering a proven solution for countries lacking the resources to manage the disease.
Despite clinical progress, challenges in accessing rare disease care persist, primarily due to cost and insurance coverage limitations.
As China has introduced an updated version of national medical insurance drug catalog and the inaugural edition of the commercial insurance innovative drug list, the lists are expected to improve access to new and innovative drugs while making commonly used medications more affordable.
Studies show that most rare disease patients in the world endure more than a five-year diagnostic delay, typically enduring seven consultations and three misdiagnoses before receiving an accurate diagnosis.
For families like that of Candy, the statistics take a backseat to a simple reality of a child who no longer needs a needle in her arm every three weeks.
"We spent years imagining a future without regular visits to the hospitals," her father shared, his heartfelt gratitude conveyed through a translator. "From now on, she will have the normal life we once thought was out of reach." ■
