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Focusing on rare dyslipidemia, building a patient support network-HoFH/FCS Patient Advocacy Workshop was successfully held in Beijing

2026-09-25 15:15

Focusing on rare dyslipidemia, building a patient support network-HoFH/FCS Patient Advocacy Workshop was successfully held in Beijing

·Preface ·

On September 18-19, 2026, the "2026 China HoFH Patient Survival Status Survey Report and HoFH/FCS Advocacy Workshop," hosted by Homy FH China Patient Network, was held as scheduled in Beijing.

This event serves a dual purpose: it serves both as a public research report release and as an advocacy workshop focused on patients' real situations.

On September 18, our launch event was successfully held, and on the 19th, we shifted the focus from "data release" to "action implementation"—focusing on the HoFH/FCS patient advocacy workshop.

PART.01

A workshop gathered for the "few."

Before officially starting the discussion, everyone played a mini-game called "Find Friends" together, breaking the ice through the game and adding a bit of familiarity to everyone.

Afterwards, experts from the clinical frontline, representatives from patient organizations, and patients' families gathered to engage in an in-depth dialogue on two rare hereditary dyslipidemia disorders: homozygous familial hypercholesterolemia (HoFH) and familial chylomicromia syndrome (FCS), covering diagnosis, treatment, management, and protection. The main theme of the entire event boiled down to two words—advocacy and connection.

PART.02

Why are HoFH and FCS
worth watching?

HoFH and FCS are two rare hereditary dyslipidemia that are often confused but belong to different pathways.

The problem with HoFH lies in the "cholesterol pathway"

Due to defects in the LDLR gene, patients cannot effectively clear LDL-C cholesterol from their bodies, exposing them to extremely high cholesterol levels from birth, and the risk of early-onset atherosclerotic cardiovascular disease is closely linked.

FCS points to the "triglyceride-chylomicron pathway"

Lipoprotein lipase (LPL) dysfunction prevents chylomicrons from being properly broken down, causingplasma triglyceride levels to rise dramatically, usually persistently above 10 mmol/L. Recurrent acute pancreatitis is the most dangerous blade hanging over the patient's head.

The common traits of these two diseases are "rare" and "severe."

The incidence of HoFH is 1/360,000 to 1/250,000;
The overall population incidence of FCS is about 1/1000000 to 2/1000000;
Both are considered rare diseases among rare diseases. Delayed diagnoses, the threat of complications, and the burden of lifelong management form the foundation of their daily lives. What they need is not just medicine, but also being seen, understood, and supported.

PART.03

Expert Perspective:
Current Status of Diagnosis and Treatment and New Therapeutic Patterns

At the event, Professor Qin Yanwen from Beijing Anzhen Hospital, Capital Medical University, delivered an academic report titled "Current Status and Drug Landscape of HoFH and FCS Diagnosis and Treatment," systematically outlining the diagnosis and treatment context of the two diseases for the patients present.

Qin Yanwen, Beijing Anzhen Hospital, Capital Medical University

HoFH: The earlier, the better; the lower, the better; the longer, the better

Professor Qin Yanwen emphasized that the new (2026) EAS Child and Adolescent FH Expert Consensus has clearly proposed a lipid-lowering strategy of "the earlier the better, the lower the better, and the longer the better."

This means:

Early screening and diagnosis are key steps to reduce cardiovascular risks for patients;
Strengthening lipid reduction by risk stratification and achieving LDL-C compliance is the primary goal of HoFH management;
Guidelines both domestically and internationally unanimously call for all suspected FH patients to undergo comprehensive medical history, family history assessment, and comprehensive laboratory examination.

FCS: Dietary therapy is an irreplaceable cornerstone of management

The report points out that the Chinese "FCS Diagnosis and Management Expert Consensus" briefing has combined European and American standards with Chinese clinical practice to formulate locally appropriate diagnostic criteria—maintaining fasting triglycerides at ≥10 mmol/L on the basis of standard lipid-lowering treatment is an important diagnostic basis.

Since conventional lipid-lowering drugs are often ineffective for FCS patients, dietary therapy has become an irreplaceable cornerstone of their management.

The treatment landscape is being rewritten

What excites patients even more is that the treatment landscape is being rewritten. Professor Qin Yanwen introducedseveral cutting-edge new drugs and novel treatment mechanisms

Medications for HoFH patients

Microsomal triglyceride transfer protein (MTP) inhibitors (such as Lometapai, launched on July 31, 2026, now available for purchase in some cities)
Inhibitors targeting ANGPTL3 (such as ivesumumab marketed abroad but not yet domestically; SHR-1918 and VSA003 have completed Phase III clinical trialsdomestically).

Medications for FCS patients
RNA interference (RNAi) therapy targeting APOC3: Prostrane sodium was launched domestically in July 2026.
Gene-edited drugs targeting APOC3 (such as Sequence Biologics' CS-1214, which completed dosing of the first adult and pediatric FCS patients)

These explorations are gradually pushing the vision of "promising medicine" from a vision into reality.

PART.04

Dietary Management:
The patient's daily "another battlefield"

If medication is the main battlefield of treatment, then dietary management is the "other battlefield" patients face every day.

Dr. Pan Changlu from the Clinical Nutrition Department at Beijing Children's Hospital, Capital Medical University, provided patients with practical explanations on managing two rare dyslipidemia diets.

Beijing Children's Hospital affiliated with Capital Medical University, Pan Changlu

Food selection recommendations

For FCS patients who need to strictly limit fat intake, Professor Pan Changlu offers specific food choices:

Foods with less than 2 grams of fat per 100 grams, such as:

Rabbit meat, lamb tenderloin, duck breast
Removing the head and skin from the shrimp
Skim milk, egg whites, soy milk, water gluten

These foods can be considered high-quality protein and are also important sources of protein. Foods with relatively high fat content should be consumed in limited amounts.

Cooking oil: Key points for using MCT oil

For cooking oils, Professor Pan Changlu especially recommends MCT oil (medium-chain fatty acids). This special vegetable oil is already available on the market in both powder and aqueous forms.

However, there are two points to note when using it:

MCT has a low smoke point and requires low-temperature cooking and storage;
After strictly limiting fat, especially for younger children, essential fatty acid deficiencies are more likely. If signs of growth retardation or dry skin appear, immediate alertness is needed.

These detailed reminders reflect the real survival pressure of FCS patients—over 90% report difficulty managing fat intake, and "a single wrong bite can trigger devastating pancreatitis pain."

For this reason, multidisciplinary management involving gastroenterologists and nutritionists is especially important for patients.

PART.05

From Demands to Action:
Patient participation in medical insurance and access protection

Drug launch is only the first step. How to ensure patients truly "use and afford it" is another topic that drew significant attention in this workshop.

Professor Guo Jinchuan from the Beijing Illness Challenge Foundation shared a special discussion titled "Patient Participation in Medical Insurance/Commercial Insurance Access: From Demands to Action."

Guo Jinchuan, Director of Information Research, Beijing Illness Challenge Foundation

The discussion centered on the real-world issues that patients care about most:

Drugs that have already been marketed have not yet been included in medical insurance. Besides medical insurance, are there other affordable alternative coverage?
Many drugs currently lack pediatric indications. Can patients participate in promoting more drugs to fill the gap in pediatric medication?
What kind of support can outpatient policies for special and chronic diseases provide for patients on long-term medication?

These issues point to the "last mile" common among rare disease patients in the era of innovative drugs.

The message conveyed by the workshop was clear:patient organizations are moving from simply "expressing demands" to actively "driving action"—by mastering the basics of medical and commercial insurance payments, they are participating in the process of promoting drug accessibility.

PART.06

Walking hand in hand, letting Hanxian live better

A workshop that connects experts, patients, and advocates.

From cutting-edge diagnosis and treatment to daily diet, from medication access to guaranteed access, consensus was built through dialogue.

Red Rice Grains Mutual Aid Home will continue to walk with fellow patients, treating every rare treat with tenderness.