Birch triterpenes

【你敢给自己一个拥抱吗？】 这个问题，对大多数人来说，根本不需要想。 双臂一环，把脸埋进胸口，再正常不过的动作。你懂的。 但对一类孩子来说——这个动作本身就是一种伤害。 他们的皮肤，脆弱得像蝴蝶翅膀。不是比喻。是真的会破。 轻轻一个拥抱，皮肤会撕裂、起泡、溃烂。换一件衣服，背上磨出一片血泡。就连走路，脚底都会磨出水疱——因为摩擦，对他们来说就是创伤。 这种病，叫大疱性表皮松解症，英文简称EB。 得了这个病的孩子，有一个美丽的名字—— "蝴蝶宝贝"。 今天，我们来讲这群特殊孩子的故事。准备好，这期内容会让你重新理解——什么是坚强。 【这种病，到底有多"脆"？】 先说说"大疱性表皮松解症"这个名字。 "大疱"，就是大水疱。"表皮松解"，就是表皮和真皮之间的连接松了、容易裂开。合起来理解：皮肤轻微摩擦就会起大疱、撕裂、溃烂。 那"蝴蝶宝贝"这个名字是怎么来的？ 因为他们的皮肤，就像蝴蝶的翅膀一样——美丽，但一碰就碎。 正常人的皮肤，表皮和真皮之间有一层"锚"，像钉子一样把两层皮肤牢牢固定在一起。你可以把它想象成皮肤里的"胶水"。 但EB患者的这个"胶水"，基因里就写错了。 结果就是：皮肤表面看起来完好无损，但只要稍微一摩擦——穿衣服、走路、甚至被人碰了一下——表皮和真皮就会分开，组织液渗出来，形成一个水疱。 更残酷的是：水疱破了之后，就是一片开放的伤口。而这些伤口，会反复感染、愈合、再破裂，永无止境。 想象一下：你全身有几十个、上百个这样的伤口，每天都在疼。 这不是比喻，这是EB患者每天的真实生活。 EB不只是皮肤的问题。因为基因突变，身体的很多部位都会受影响： • 口腔黏膜：吃东西会磨出口疱，很多EB患者吃饭本身就是一种折磨。严重的人，只能吃流食。 • 食道：食管也会起疱、狭窄，吞咽食物变得极其困难。 • 眼睛：角膜会反复受伤，很多EB患者视力下降，甚至失明。 • 手指：反复起疱、愈合，导致手指皮肤挛缩、融合，很多患者手指逐渐"并指"，失去抓握能力。 • 指甲：很多EB患者根本没有指甲，或者指甲反复脱落。 【基因里的一行错代码】 EB是一种遗传病，也就是说——从出生那天起，基因里就带着这个缺陷。 人的皮肤之所以能扛住摩擦，靠的是几种关键的"结构蛋白"，它们就像皮肤里的"钢筋"： • 胶原蛋白VII型（COL7A1基因）：它形成"锚原纤维"，把表皮和真皮铆在一起。这个基因出问题，就是营养不良型EB（DEB）。 • 层粘连蛋白332（LAMB3等基因）：它位于"基膜"，是表皮和真皮之间的"双面胶"。这个基因出问题，就是交界型EB（JEB）。 • 角蛋白5或14（KRT5、KRT14基因）：它们构成表皮基底细胞的"骨架"。这个基因出问题，就是单纯型EB（EBS）——这是最轻的一型。 根据基因突变的位置不同，EB主要分四大类： 1. 单纯型EB（EBS）——最轻 水疱主要在表皮浅层，不怎么会留疤。很多患者成年后症状会减轻。你可以理解为"皮肤敏感加强版"。 2. 交界型EB（JEB）——最凶险 水疱在表皮和真皮的交界处，这个类型的患儿，很多在出生不久就去世了。特别是"Herlitz型"，非常凶险。但幸运的是，这一型比较罕见。 3. 营养不良型EB（DEB）——最常见，也最痛苦 水疱在真皮深层，愈合后会留疤、挛缩。这一型的患者，手指会逐渐融合成"连指"，嘴巴会因为瘢痕挛缩而张不开（称为"假性并指"和"小口症"）。更可怕的是，长期慢性伤口会癌变，很多DEB患者成年后会得皮肤癌。 4. Kindler综合征——最复杂 这是一种非常罕见的类型，除了皮肤脆弱，还会伴有光敏感、皮肤萎缩等多种问题。 发病率有多高？ 活产儿中，大约每10万个新生儿里有2个得EB。 听起来好像不多？但你要知道，中国每年出生约1000万新生儿，这意味着每年有200个左右的孩子一出生就被诊断为"蝴蝶宝贝"。 目前中国约有2-3万名EB患者，全球约有50万人。每一个数字背后，都是一个家庭十几年的坚持。 【三个蝴蝶宝贝的真实人生】 ▼ 第一个故事：高尚好，25岁的生命奇迹 ▼ 2001年3月，高尚好出生在河南郑州。 出生那天，医生看到她的腿——皮肤缺失了一大块，右眼也因为水疱溃烂而失明了。医生对她的父母说了一句话： "这个孩子，可能活不过两岁。" 这句话，高尚好的父母记了一辈子。 高尚好在ICU里待了166天。166个日夜，她的父母守在保温箱外面，看着她身上缠满纱布，看着她因为疼痛而哭到没声。 她活过了两岁。又活过了五岁。十岁。十五岁。 但你以为这就算熬出头了？不。 13岁那年，高尚好进入了青春期。疾病带来的痛苦、外表的异样、同学们异样的眼光，让她彻底崩溃了。她拒绝锻炼、拒绝护理，整个人的身体功能迅速倒退——手指挛缩得更厉害了，腿也弯成了90度，没法走路。 那段时间，她每天躺在床上，看着天花板，问妈妈："为什么是我？我做错了什么？" 妈妈没有回答。她也不知道怎么回答。她只能每天早上帮女儿换药，晚上帮女儿翻身，日复一日。 后来，高尚好做了好几次分指手术，把融合在一起的手指重新分开。又做了膝部牵拉训练，把弯成90度的腿一点点拉直。每一次训练，都疼到浑身发抖。 现在的她，即将迎来25岁生日。 身高1.5米，体重30公斤。右眼失明了。手指弯曲。但她活着，而且活得有尊严。 她每天要换三次药，每次大换药要花3个小时以上。妈妈会把每一处伤口都拍照存档，建立一个"伤口愈合曲线"，方便医生远程指导。 吃饭要细嚼1个小时，因为口腔里也有水疱，每嚼一口都疼。但她坚持自己吃，不靠别人喂。 她说："我不想让别人觉得，我是一个废物。" ▼ 第二个故事：罗仁心，16岁的"瓷娃娃"？不，是蝴蝶宝贝 ▼ 2025年10月，贵州。16岁的罗仁心，身高128厘米，体重21公斤。 因为EB，他的生长发育严重受限。骨骼发育不好，个子长不高。皮肤一碰就破，手掌已经因为反复起疱、愈合而完全变形——他"失去了手掌"，那只手现在看起来就像一个拳头。 但他有一个梦想：他想上学。 因为身体原因，罗仁心一直没法正常上学。但他自己在家自学，认字、算数、读课外书。他说："我很想上学，想和别的小孩一样，坐在教室里。" 记者去采访他的时候，他坐在特制的软垫上，用变形的手指捧着一本书，眼睛亮亮的。 他说："虽然我的皮肤会破，但我的心不会。" ▼ 第三个故事：王子超，用镜头记录蝴蝶宝贝的人 ▼ 王子超，江苏南通人，也是一名EB患者。但他的故事有点不一样——他没有停留在"被帮助"的角色，而是成为了"帮助者"。 因为疾病，他的手指已经并指挛缩，丧失了基本劳动能力。但他学会了用相机。 2024年，他宣布要拍一部纪录片，记录中国EB患者的真实生活。他走遍全国，去采访其他蝴蝶宝贝，记录他们的日常、他们的痛苦、他们的坚持。 每次外出拍摄，对王子超和妻子来说，都是身体和意志的双重考验。他的皮肤会在路上摩擦起疱，他要一边换药一边拍。 有人问他："你自己都这样了，为什么还要去拍别人？" 他说："因为我想让更多人知道，蝴蝶宝贝不是'怪胎'，他们和我们一样，有自己的梦想，有自己的尊严。" 2025年3月，这部纪录片发布了。片子里，有各种各样EB患者的面孔——有孩子，有成年人，有还在坚持上学的，有已经为人父母的。 王子超用他变形的手指，按下快门，记录下这些面孔。他说："我们每个人的皮肤都碎过无数次，但我们的心，从来没有碎过。" 【希望在哪儿？三道曙光】 讲完三个故事，你可能会觉得沉重。但我想告诉你——现在，EB的治疗，正在迎来前所未有的突破。 ★ 第一道曙光：第一款"对症药"终于来了 2023年12月，美国FDA批准了一款药——Filsuvez（中文名：桦醇三萜凝胶）。这是全球第一个专门用于治疗EB相关伤口的药物。 它的原理是什么？简单来说，它是一种从桦树皮里提取的天然成分制成的凝胶，能够： • 促进伤口愈合，缩短愈合时间 • 减轻换药时的疼痛 • 减少伤口渗液 在规模最大的全球三期临床试验中，结果显示： • 治疗组在45天内伤口愈合的几率，比对照组提高了约44%。 • 目标创面的平均闭合时间，缩短了6.8天。 • 最重要的是：它减少了换药频率。以前EB患儿每天要花2-4个小时换药，现在这个时间缩短了。 2025年10月，这款药依托海南博鳌乐城"特许使用"政策，已经在中国落地了。由复旦大学附属儿科医院李明教授团队和海南省妇女儿童医学中心黄晓燕教授团队共同诊治。 目前，这款药的上市注册申请正在加速进行中，力争尽早在中国全面获批。目标是：让患者"买得上、用得好、付得起"。 ★ 第二道曙光：基因治疗，一次性"修好"基因缺陷 如果说Filsuvez是"治标"——促进伤口愈合，那么基因治疗就是"治本"——直接修复导致EB的基因突变。 目前，有几个方向非常值得关注： • 斯坦福医学院的研究团队，开发了一种用"基因修饰的皮肤移植"治疗EB的方法。他们把患者的一小块皮肤取出来，在实验室里用基因疗法"修好"突变的基因，然后再移植回患者身上。这些"修好"的皮肤，不再起疱了。 • 2026年2月，斯坦福医学院发表在《Stanford Medicine》杂志上的研究显示：他们开发了两种基因疗法，用于治疗这种罕见的皮肤病。FDA已经在2025年批准了其中一种基因疗法皮肤移植用于EB患者。 • 更前沿的：CRISPR基因编辑技术结合纳米颗粒"导航车"技术，已经在皮肤模型中修复了近30%的EB相关目标基因缺陷。虽然还在实验室阶段，但为将来的临床应用铺了路。 你可以这样理解：以前，EB患者的基因里有一行"错代码"，医生束手无策。现在，科学家可以直接"改写"这行代码，让皮肤恢复正常。 这不是科幻。这正在发生。 ★ 第三道曙光：AI加速新药研发，让"等药"不再遥遥无期 传统新药研发要10-15年。但对EB患者来说，他们等不起。 现在，AI正在改变这个局面。 • AI可以模拟数亿种化合物，找出最可能有效的那一个，把筛选时间从几年缩短到几个月。 • AI可以预测蛋白质结构（你可能听说过AlphaFold），帮助科学家设计更精准的基因疗法。 • 更重要的是：AI可以帮助"repurposing"——也就是发现已有药物的新用途。说不定，某个已经上市的药，恰好能治EB。AI可以在短时间内筛查完所有已上市药物，找到这样的"惊喜"。 2025-2026年，有多款EB基因疗法进入临床试验阶段。如果一切顺利，未来5-10年内，我们可能会看到第一款"一次性治愈"EB的基因疗法获批。 到那时候，"蝴蝶宝贝"这个名词，可能就会成为历史。 【写在最后：脆弱的力量】 讲到这里，我想说几句心里话。 蝴蝶宝贝们，他们的皮肤脆弱得像蝴蝶翅膀，一碰就碎。但他们的生命，却比很多人都要坚韧。 高尚好，被告知活不过两岁，现在快25岁了。她说："我不想让别人觉得，我是一个废物。" 罗仁心，16岁，身高128厘米，体重21公斤，手掌已经变形。但他说："虽然我的皮肤会破，但我的心不会。" 王子超，手指并指挛缩，丧失了基本劳动能力。但他扛起相机，走遍全国，记录其他蝴蝶宝贝的故事。他说："我们的心，从来没有碎过。" 瓷器易碎，但瓷器可以传世千年。 蝴蝶的翅膀一碰就碎，但蝴蝶可以飞越千山万水。 这些孩子们，他们用自己脆弱的身体，教会了我们一件事—— 真正强大的力量，不是刀枪不入，而是——就算碎了一万次，也要一万零一次站起来。

Priority review recognizes the serious nature of epidermolysis bullosa (EB) and the urgent need for continued evaluation of potential treatment options If approved, FILSUVEZ has the potential to become the first therapy approved by Health Canada specifically for the treatment of wounds associated with dystrophic and junctional epidermolysis bullosa With the establishment of Chiesi Canada as an affiliate of Chiesi Group in January 2026, Chiesi Canada aims to support people living with rare diseases across Canada as part of the Company's legacy of care spanning generations PARMA, Italy and WOODBRIDGE, Ontario, June 09, 2026 (GLOBE NEWSWIRE) -- Chiesi Global Rare Diseases, a business unit of Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, today announced that Health Canada has accepted the New Drug Submission (NDS) for FILSUVEZ ® (birch triterpenes) topical gel for priority review for the proposed indication of the treatment of wounds associated with dystrophic and junctional epidermolysis bullosa in patients 6 months of age and older. Under Health Canada’s priority review pathway, the NDS is subject to an accelerated review timeline of approximately 180 days, in comparison to the traditional 300-day timeline. Epidermolysis bullosa (EB) is a rare genetic disorder characterized by extremely fragile skin, chronic wounds, and significant pain, which can be a substantial burden on both patients and caregivers. Health Canada grants Priority Review to therapies intended to treat, prevent, or diagnose serious or life-threatening conditions where there are limited or no comparable treatment options, or where the therapy offers a meaningful improvement over existing care. This designation reflects Health Canada’s determination that the submission meets the criteria for accelerated review in an area of serious medical need for people living with EB in Canada. “Priority Review acceptance by Health Canada marks an important regulatory milestone in the review for FILSUVEZ in Canada for people living with this rare, genetic, and often debilitating condition,” said Mitch Goldman, Senior Vice President, R&D, Chiesi Global Rare Diseases. “EB has historically been characterized by limited research and treatment options, but the field is evolving, and so are we. With our history in Canada as a center for research and development, and now as an affiliate, we are proud to contribute to progress for the community and privileged to partner with local regulators, patients, caregivers, and clinicians. We are hopeful that this milestone brings us closer to our goal of advancing rare disease care in Canada and supporting those living with EB.” “For individuals and families living with EB, everyday life can involve significant challenges, from managing chronic pain to undertaking complex and time-intensive wound care,” said Erin Hoyos, Executive Director, DEBRA Canada . “Health Canada’s Priority Review of FILSUVEZ is an important milestone for the EB community and reflects continued progress in the evaluation of potential treatment options for people living with this condition in Canada.” “Health Canada’s acceptance of this submission for Priority Review of FILSUVEZ highlights the importance of continued scientific advancement and additional therapeutic options for the EB community,” said Dr. Irene Lara-Corrales, MD, MSc, Paediatric Dermatologist at The Hospital for Sick Children (SickKids) in Toronto . “Epidermolysis bullosa remains highly challenging to manage, with patients often experiencing recurrent wounds, pain, infections, and other serious complications throughout their lives. The disease can also place a substantial physical, emotional, and caregiving burden on patients and families, often requiring ongoing multidisciplinary care and support. An approved therapy in Canada for individuals living with EB would represent an important potential advancement for the community.” The submission is supported by data from the pivotal Phase 3 trial, EASE, which was published in the British Journal of Dermatology in 2023, that assessed the safety and efficacy of FILSUVEZ in patients with dystrophic and junctional epidermolysis bullosa, with a 24-month open-label follow-up period. 1 ,2 The safety and efficacy of FILSUVEZ for the proposed indication remain under review by Health Canada, and FILSUVEZ has not received market authorization in Canada. FILSUVEZ has been approved by the U.S. Food and Drug Administration (2023) and the European Commission (2022) for the treatment of wounds associated with dystrophic and junctional EB. FILSUVEZ was officially registered by the Australian Register of Therapeutic Goods (ARTG) on May 5, 2026. About Epidermolysis Bullosa Epidermolysis bullosa (EB) is a rare genetic disorder characterized by extreme skin fragility and blistering. Severe forms lead to chronic blisters, ulcerations, scarring, joint contractures, esophageal strictures, a high risk of squamous cell carcinoma, infections and premature death. 3 Epidermolysis Bullosa is a group of rare, inherited skin fragility disorders in which the skin is very weak and fragile. Even minor friction to the skin can cause painful blisters and open wounds that are slow to heal and can lead to significant scarring. 3 Not only does EB affect the external skin, but the lining of the mouth, throat, eyes, and the digestive system. 4 There are four main types of EB and they range in how severely they affect a person. The most severe forms are junctional EB (JEB), and dystrophic EB (DEB). These severe EB subtypes may be accompanied by multiple complications (e.g. sepsis, failure to thrive, severe anemia, cancer) leading to a reduced lifespan. People with EB must take extraordinary measures to protect their fragile skin, and despite their efforts, many live with chronic wounds all over their body. These wounds are extremely painful and itchy. 4 Pain can be persistent and severe in EB patients. 5 Globally, an estimated 500,000 people are living with EB. 6 About EASE Trial The EASE Study was a Phase III, double-blind, randomized, vehicle-controlled trial conducted to assess a potential treatment for inherited forms of EB, including dystrophic and junctional subtypes. The subjects were randomized and treated for three months in a double-blind manner with either Oleogel-S10 (birch triterpenes) or a placebo gel consisting of refined sunflower oil, yellow beeswax and carnauba wax. The respective gel was applied in a 1 mm thick layer to all wounds each time the wound dressing was changed (every 1 to 4 days). A superficial wound with an area of 10 to 50 cm 2 was selected as the target wound by the investigator at the start of the study. This wound was used to determine the primary efficacy endpoint: the proportion of patients in whom the target wound had completely closed for the first time by day 45. After completion of the double-blind phase of the study, it was continued as an open-label study, and all patients were treated with Oleogel-S10. The primary efficacy endpoint was achieved in 41.3 percent of patients in the verum group and in 28.9 percent of patients in the placebo group. Side effects occurred with similar frequency in both comparison groups and were mostly mild to moderate. The most frequently reported treatment-related AE was wound complication (13.8% in the Filsuvez group and 14.9% in the placebo group). 1 , 7 , 8 About Chiesi Group Chiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company's mission is to improve people's quality of life and act responsibly towards both the community and the environment. By changing its legal status to a Benefit Corporation in Italy, the US, France and Colombia, Chiesi's commitment to creating shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, Chiesi is part of a global community of businesses that meet high standards of social and environmental impact. The company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035. With 90 years of experience, Chiesi is headquartered in Parma (Italy), with 31 affiliates worldwide, and counts more than 7,500 employees. The Group's research and development center in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden. For more information visit About Chiesi Global Rare Diseases Chiesi Global Rare Diseases is a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases. As a family business, Chiesi Group strives to create a world where it is common to have a therapy for all diseases and acts as a force for good, for society and the planet. The goal of the Global Rare Diseases unit is to ensure equal access so as many people as possible can experience their most fulfilling life. The unit collaborates with the rare disease community around the globe to bring voice to underserved people in the health care system. For more information visit . Follow @ChiesiGlobalRareDiseases on LinkedIn , Facebook , Instagram , X and YouTube . Chiesi Global Rare Diseases Media Contact Sky Striar LifeSci Communications Email: sstriar@lifescicomms.com References 1) Kern JS, Sprecher E, Fernandez MF, Schauer F, Bodemer C, Cunningham T, Löwe S, Davis C, Sumeray M, Bruckner AL, Murrell DF; EASE investigators. Efficacy and safety of Oleogel-S10 (birch triterpenes) for epidermolysis bullosa: results from the phase III randomized double-blind phase of the EASE study . Br J Dermatol. 2023 Jan 23;188(1):12-21. doi: 10.1093/bjd/ljac001. PMID: 36689495. 2) Fine, J.-D., Johnson, L. B., Weiner, M., Suchindran, C., & the Epidermolysis Bullosa Clinical Characterization and Outcomes Database Investigators. (2023). Inherited epidermolysis bullosa: Updated recommendations on diagnosis and classification . British Journal of Dermatology, 188 (1), 12–18. 3) Denyer J, Pillay E, Clapham J. Best practice guidelines for skin and wound care in epidermolysis bullosa. An International Consensus. Wounds International, 2017 4) Fine JD. Inherited epidermolysis bullosa. Orphanet Journal of Rare Diseases2010; 5(12): doi.org/10.1186/1750-1172-5-12. 5 5) Tabor A, Pergolizzi JV, Marti G, et al. Raising awareness among healthcare providers about Epidermolysis Bullosa and advancing toward a cure. J ClinAesthet Dermatol 2017; 10(5): 36-48 6) Epidermolysis Bullosa Research Partnership. “What Is EB?” EB Research Partnership, 7) Murrell DF, Bodemer C, Bruckner AL, Cunningham T, Davis C, Fernández MF, Kiritsi D, Maher L, Sprecher E, Torres-Pradilla M, Kern JS. Long-term safety and efficacy of Oleogel-S10 (birch bark extract) in epidermolysis bullosa: 24-month results from the phase III EASE study. Br J Dermatol. 2025 May 19;192(6):1007-1017. doi: 10.1093/bjd/ljaf022. PMID: 39821055. 8) Data on File, Chiesi Global Rare Diseases, EASE Study CSR, March 2021. FSVEZ/CA/001