LHON Patients from Japan & Myanmar Find Treatment at Lecheng

Patient Profiles

Patient A: 22-year-old male from Myanmar. First-time traveler to China, discovered Lecheng through an online search.

Patient B: 54-year-old male from Japan. Frequent traveler to China, referred by friends and family.

Diagnosis

Both patients were diagnosed with Leber Hereditary Optic Neuropathy (LHON) – a mitochondrial genetic disorder that causes rapid, progressive vision loss. LHON is a maternally inherited rare disease, with males 3 to 5 times more likely to be affected than females. Globally, it affects approximately 1 in 15,000 to 50,000 people. In China alone, an estimated 126,000 individuals are living with this condition.

Challenge

For decades, there was no targeted therapy for LHON. Before Ruijin Hainan Hospital introduced idebenone (150mg tablets), patients in China, Japan, Myanmar, and neighboring countries lacked access to any standardized treatment. Local patients could only rely on nutritional supplements and basic physical therapy – interventions that cannot fundamentally halt the ongoing damage to the optic nerve. Vision loss typically progresses relentlessly, with many patients facing legal blindness within months of onset.

The 22-year-old from Myanmar had consulted doctors in his home country and neighboring Thailand. The answer was always the same: no cure, no treatment. He discovered Lecheng through a desperate online search. The 54-year-old Japanese patient, a frequent traveler to China, learned about the treatment through friends and family. Both were running out of time – and out of options.

Lecheng Solution

Through the Boao Lecheng Pilot Zone’s “first-pilot, first-use” policy, idebenone (150mg tablets) was granted special access for LHON treatment – making it available at Ruijin Hainan Hospital before full national approval.

The treatment was delivered by a multidisciplinary team: remote support from Dr. Shen Xi (Chief Ophthalmologist, Ruijin Hospital Shanghai) and Dr. Zeng Zhaoxia (Zhongshan Ophthalmic Center), with on-the-ground execution by Dr. Wang Xu and his team at Ruijin Hainan Hospital. The team conducted a comprehensive evaluation of each patient’s genetic subtype, disease duration, and extent of visual impairment – confirming that both patients were within the therapeutic window for idebenone treatment.

Idebenone is not a cure, but a stabilizer – a drug that can slow or halt the progression of vision loss, preserving remaining sight and, in some cases, allowing for partial recovery.

Results

Both patients tolerated the treatment well. They returned home with something they had not had in months: genuine hope for preserving their vision.

Since idebenone became available, Ruijin Hainan Hospital has treated nearly 100 LHON patients from China and abroad. The hospital has become a regional hub for rare disease care, attracting patients from across East and Southeast Asia – countries where the drug remains otherwise unavailable. The successful treatment of these two international patients marks a significant step in establishing Ruijin Hainan Hospital as a specialized rare disease service window for the Asia-Pacific region.