Loose skin, wrinkled face, rigid joints, thin hair, hoarse voice, and weak body are symptoms often observed in elderly people. However, they are occurring in some innocent children. These children not only experience premature aging on the outside, but also go through a rapid aging in all bodily functions.
Kang Kang (assumed name) is one of these children. When he was a baby, he began to show these senile symptoms, especially in his feet. Due to rigid foot bones, his ankles could not move normally, and he could barely walk on his heels. After being diagnosed with Hutchinson-Gilford Progeria Syndrome (HGPS), a fatal rare disease with an incidence of one in four to eight million, Kang Kang seemed to have started the countdown of his life.
Currently, there are more than 7,000 confirmed rare diseases worldwide. As an extremely rare and fatal genetic disease among rare diseases, HGPS is reported in about 60 to 70 people in China. Children with HGPS age five to ten times faster than normal children physically, and in body organs. In most circumstances, they die of arteriosclerosis or other cardiac events at about 14.5. Currently, targeted drug therapy is the only therapy that has proved effective against HGPS.
On the afternoon of April 22, the launching ceremony of the “Relay for Life– Public Welfare Program for Treatment of Children with HGPS” was successfully held at the Lakeside Branch of the Children’s Hospital of Zhejiang University School of Medicine. Ms. Wang Qi, General Manager of Zhejiang Medical Technology Development Co., Ltd. (a wholly-owned subsidiary of Hong Kong Winhealth Pharma Group), attended the launching ceremony and the subsequent donation ceremony as a donor representative.
Scene of launching ceremony
The program is directed by Zhejiang Medical Products Administration, Health Commission of Zhejiang Province, and Zhejiang Women's Federation, and co-sponsored by the Children’s Hospital of Zhejiang University School of Medicine, Zhejiang Women and Children’s Foundation, and the Chinese Organization for Rare Disorders (CORD). By offering drugs and care services intended to treat HGPS, the program aims at extending the life and alleviating the symptoms of children with HGPS.
Group photo taken at the donation ceremony
Witnessed by all parties concerned, the “Relay for Life– Public Welfare Program for Treatment of Children with HGPS” was launched by the representatives at the scene, including Mao Jianhua, Deputy Director of the Children’s Hospital of Zhejiang University School of Medicine; Feng Zhigang, Secretary General of Zhejiang Women and Children’s Foundation; Huang Rufang, founder of the Chinese Organization for Rare Disorders; and Wang Qi, General Manager of Zhejiang Medical Technology Development Co., Ltd.
It should be noted that the preparations for the “Public Welfare Program for Treatment of Children with HGPS” first started at the end of 2019. After two years, it was formally launched with the substantial support and great efforts of all parties concerned.
During drug import, Winhealth Pharma actively coordinated with upstream manufacturers on product supply, and handled the import of Lonafarnib, a drug urgently needed in clinical practice on their behalf. Adhering to the original intention of “patient comes first”, Winhealth Pharma has overcome a series of pandemic and logistics difficulties relying on their professional competence and active communication, and delivered the first batch of Lonafarnib donated by Progeria Research Foundation (PRF) and Eiger BioPharmaceuticals to the hospital. Two children with HGPS (Kang Kang included) have arrived at the Children’s Hospital of Zhejiang University School of Medicine this week for formal drug therapy.
Lonafarnib is a symptomatic drug that has been approved by FDA for treatment of “HGPS”. However, as the drug is very expensive and has not been approved for marketing in China, how to make medication available to patients has always been a difficulty faced by experts and patients' families. The launching of this program has, undoubtedly, brought great hope and encouragement to patients and their families.
Visiting children with HGPS at the ward
After the ceremony, the guests met Kang Kang and the other child in the ward and gave them flowers, nourishments, and toys, hoping that they would stay strong and optimistic during treatment at the hospital. A family member of Kang Kang said that, "It is unfortunate that our child has had this rare disease, but we are also lucky to receive timely treatment, thanks to our country, doctors , and experts, as well as related foundations, units, and people from all walks of life!”
At present, a large number of patients with rare diseases in China are still faced with the dilemma of drugs available at abroad but not in China. The use of this special drug in Zhejiang is a new breakthrough, which brings hope to patients who are eager for drug therapy. In the future, the pace of treatment will not stop, and the new hope will continue to grow! It is our sincere belief that, with the concerted efforts of our country and all sectors of society, patients with rare diseases will embrace a brighter future.
Liu Zhihong, Academician of Chinese Academy of Engineering, Director of the National Clinical Research Center for Kidney Disease, the General Hospital of Eastern Theater Command, and Directory of Zhejiang University School of Medicine
Zhejiang Medical Products Administration
Health Commission of Zhejiang Province
Children’s Hospital of Zhejiang University School of Medicine
Zhejiang Women and Children’s Foundation
Chinese Organization for Rare Disorders (CORD)
Hangzhou Market Supervision Administration
Rare Diseases Branch of Zhejiang Medical Association
Progeria Research Foundation (PRF)
Comprehensive Business Section III of Qianjiang Customs
Hangzhou Xiaoshan International Airport Customs