Medical Care in China

Amyotrophic Lateral Sclerosis (ALS)
Treatment in China

Provide a clear and complete coordinated pathway from expert assessment to treatment and rehabilitation.

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Multidisciplinary consultationsinvolving joint analysis of complex cases by different specialties.
Accurately identify the cause of the diseaseby combining imaging, laboratory and genetic testing.
Tackling complex casesFocusing on Relapsed, Refractory, and Poorly Treated Cases.
Personalized plans aredeveloped based on the patient's actual condition to create a treatment pathway.

Condition Guide

About Amyotrophic Lateral Sclerosis (ALS)

Understand the condition, common triggers and treatment options that may require specialist assessment.

Disease Introduction

Amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig's disease, is a neurodegenerative disease characterized by the progressive degeneration of upper and lower motor neurons that control voluntary movement. Patients experience weakness in the limbs, muscle atrophy, and difficulty swallowing and breathing, but their consciousness, thinking, and sensation are usually clear. Most patients die from respiratory failure within 3-5 years of onset, but there is significant individual variation. Early diagnosis, standardized medication, and multidisciplinary support can prolong survival and improve quality of life.


Cutting-edge high-precision diagnostic and treatment technologies

  1. SOD1 gene targeting : Tofersen is an antisense oligonucleotide intrathecal injection drug. In April 2023, it received accelerated approval from the FDA for SOD1 gene mutant ALS, and it has also been approved for marketing in China.
  2. Long-term follow-up benefits : In December 2025, JAMA Neurology published the long-term follow-up results of VALOR: the risk of death or permanent ventilation was reduced by about 36% in the early medication group compared with the delayed medication group, and the risk of death was reduced by about 48%.
  3. iPSC cell transplantation : In September 2025, Peking University Third Hospital led the initiation of a Phase I/II registration clinical trial for the treatment of ALS with iPSC-derived motor progenitor cells (XS228 cell injection) and completed the first lumbar puncture administration. This is a trial that has been submitted and approved by both China and the United States.
  4. Domestic gene therapy : In March 2025, Fan Dongsheng's team launched a multi-center IIT study of SNUG01 gene therapy at Peking University Third Hospital; other gene therapy pipelines such as RJK002 are also being promoted in China, bringing new pathways for familial ALS.
  5. Oral dosage form innovation : Edaravone sublingual tablets will be included in the National Medical Insurance Drug Catalog adjustment application in 2025. Oral/sublingual dosage forms significantly simplify the administration route and improve compliance compared to long-term intravenous infusion.

Care Overview

Amyotrophic Lateral Sclerosis (ALS) Treatment in China

The medical team will develop a personalized protocol based on the pathology type, stage, genetic test, previous treatment history, and the patient's physical condition.

Leading Specialists

Trusted Expertise Across Leading Specialties

Wanping

Associate Chief Physician, Liver Surgery Expert at Renji Hospital.

Rare & Complex Diseases

Research Areas : Pediatric liver transplantation, Kasai procedure for biliary atresia, and complex liver tumor resection, among other pediatric liver surgeries. Cutting-Edge Techniques: Proficient in the surgical treatment of pediatric liver diseases such as biliary atresia, inherited metabolic diseases, cholestasis, and hepatoblastoma, having treated over 1700 cases of various pediatric liver diseases and rare diseases; as a core member of the team, systematically conducting liver transplantation surgery

West China Hospital of Sichuan University logo Affiliated Hospital West China Hospital of Sichuan University
Wu Yu

Chief Physician, Doctoral Supervisor, specializing in rare hematological diseases

Rare & Complex Diseases

Research areas : rare hematological diseases, hereditary hemolytic diseases, systemic amyloidosis - Cutting-edge technologies: Stratified treatment for systemic amyloidosis, molecular diagnostics for complex hematological and rare diseases. - Areas of expertise: Systemic AL amyloidosis, paroxysmal nocturnal hemoglobinuria, hereditary platelet disorders - Academic Highlights: Core expert in multidisciplinary diagnosis and treatment of amyloidosis in China; Core Value: Establishment of individualized drug a

West China Hospital of Sichuan University logo Affiliated Hospital West China Hospital of Sichuan University
Zhang Shuyang

President of Peking Union Medical College Hospital, and concurrently Vice President of the Chinese Academy of Medical Sciences and Peking Union Medical College; Chief Physician, Dr., and Doctoral Supervisor

Rare & Complex Diseases

Deputy Director of the State Key Laboratory of Rare and Complex Diseases; National Health Commission Outstanding Young and Middle-aged Expert, State Council Special Government Allowance Expert, and Changjiang Scholar Dr. appointed by the Ministry of Education. Currently serves as Chairman of the Rare Diseases Branch of the Chinese Medical Association, Chairman of the Rare Disease Diagnosis and Treatment and Guarantee Committee of the National Health Commission, Director of the National Rare Disease Qualit

Peking Union Medical College Hospital, Chinese Academy of Medical Sciences logo Affiliated Hospital Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
Shen Min

Doctor of Medicine, Director of the Department of Rare Diseases, Chief Physician of Rheumatology and Immunology, Dr., and Doctoral Supervisor at Peking Union Medical College Hospital.

Rare & Complex DiseasesInternal Medicine

Visiting Scholar at the University of California, San Francisco, and Visiting Researcher in Rheumatology and Immunology at the Cleveland Clinic. Editorial Board Member of the *Chinese Journal of Clinical Immunology and Allergy*, *Chinese Journal of General Practitioners*, and *Peking Union Medical College Journal*. Vice Chairman of the Immunodeficiency Monitoring and Translation Professional Committee of the China Association for the Promotion of Human Health Science and Technology; Standing Committee Mem

Peking Union Medical College Hospital, Chinese Academy of Medical Sciences logo Affiliated Hospital Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
Zhang Guojun

Chief Physician, Dr., and Doctoral Supervisor. Currently serves as Director of the Neurology Center, Epilepsy Center, Rare Disease Center, and Neurological Diseases and Cognitive Research Laboratory at the National Children's Medical Center and Beijing Children's Hospital, Capital Medical University.

NeurologyRare & Complex Diseases

Has extensive clinical experience in the surgical treatment of functional neurosurgical diseases, especially refractory epilepsy, and has long been engaged in research on the pathogenesis and intervention strategies of epilepsy and cognitive developmental disorders. Graduated from the Department of Medicine, Hebei Medical University in 1989, and received a Ph.D. in Surgery (Neurosurgery) from Capital Medical University in 2003. Has undertaken more than ten national and provincial-level research projects a

Beijing Children's Hospital (School of Pediatrics, National Children's Medical Center), Capital Medical University logo Affiliated Hospital Beijing Children's Hospital (School of Pediatrics, National Children's Medical Center), Capital Medical University

Patient Stories

See the Next Step Through Patient Journeys

Adult Still's Disease : The End of 120 Days of CASE 01
PATIENT JOURNEY

Adult Still's Disease : The End of 120 Days of "Unexplained High Fever": How a British Pilot Regains His Career in China

Rare & Complex Diseases

Patient records Name: James (pseudonym) Age: 35 Nationality: British Diagnosis: Adult-onset Still's disease (AOSD), characterized by a triad of high fever, rash, and arthritis, accompanied by severe ferritinemia. Treatment Hospital: Department of Rheumatology and Immunology, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine For a pilot, any undiagnosed illness means the end of his career. James' nightmare began with what seemed like a common cold, but turned into a 120-day ord

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IgG4-related disease (involving the pancreas, submandibular gland, and retroperitoneal lymph nodes) - Two years shrouded in CASE 02
PATIENT JOURNEY

IgG4-related disease (involving the pancreas, submandibular gland, and retroperitoneal lymph nodes) - Two years shrouded in "tumor": How a Canadian patient overturned a wrong diagnosis in China.

Rare & Complex Diseases

Patient records Name: Margaret (pseudonym) Age: 61 Nationality: Canadian Diagnosis: IgG4-related disease (involving the pancreas, submandibular gland, and retroperitoneal lymph nodes), previously misdiagnosed as pancreatic cancer. Treatment Hospital: Department of Rheumatology and Immunology, Peking Union Medical College Hospital Case Theme: In the world of intractable diseases, the most agonizing part is often not the treatment itself, but the journey further and further down the path of treatment with a

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