我刚满25岁。患有杜氏肌营养不良症（DMD）的人通常很难再迎来下一个生日。DMD是一种进行性疾病；我的肌肉会随着时间推移逐渐萎缩，曾经轻而易举就能完成的事情，现在要么需要提前规划、寻求协助，要么根本无法再完成。甚至连睡觉都变得困难，褥疮也成了现在的问题。维持基本的生理功能需要付出大多数人从未考虑过的努力。
I just turned 25. People with Duchenne muscular dystrophy (DMD) aren't expected to see another birthday after this one. DMD is a progressive disease; my muscles weaken over time and things that used to be automatic now take planning, assistance, or simply aren't possible anymore. Even sleeping can be hard. Bed sores are now an issue. Basic physical function takes work most people never have to think about.
我知道统计数据是怎么说的。但我还没准备好让这些数据来书写我人生的结局。
I know what the statistics say. I am not ready to let them write the ending of my story.
特朗普提名的FDA局长人选在激烈的交锋中多次回避桑德斯关于全民医保的要求。我想发展我的事业，我想结婚并组建家庭。我希望几十年后醒来时，能意识到25岁并不是终点的开始，而仅仅是我有幸迎来的又一个生日。
TRUMP FDA PICK REPEATEDLY SIDESTEPS SANDERS' UNIVERSAL HEALTHCARE DEMANDS IN HEATED CLASH I want to grow my career, I want to get married and start a family. I want to wake up decades from now and realize that turning 25 wasn't the beginning of the end — it was just another birthday I was blessed enough to reach.
正因如此，我比大多数25岁的年轻人更密切地关注着参议院对FDA局长提名人海蒂·奥弗顿（Heidi Overton）博士的确认听证会。
That's why I'm watching the Senate confirmation fight over Dr. Heidi Overton, the nominee to lead the FDA, more closely than most 25-year-olds ever watch Washington.
对于大多数美国人来说，FDA局长只是新闻里的一个名字。但对于像我这样患有罕见病的3000万美国人来说，这个人决定了我有多少种治疗选择，以及我还有多少时间去尝试这些选择。
For most Americans, an FDA commissioner is just another name in the news. For the 30 million Americans like me living with a rare disease, that person can decide how many options I have and how much time I have to use them.
需要明确的是：我并不是要求FDA放弃科学。我尊重医生、临床试验和确凿的证据。我知道有些药物无效，有些药物确实存在风险。
To be clear: I'm not asking the FDA to abandon science. I respect doctors, clinical trials and hard evidence. I know some drugs don't work and some carry real risk.
但华盛顿很少问这样一个问题：究竟由谁来决定患者愿意承担多大的风险？
But there's a question Washington doesn't ask often enough: who gets to decide how much risk a patient is willing to accept?
如果我了解风险，我的医生也了解风险，而且有确凿的证据表明某种疗法可能有效，那么为什么华盛顿的监管机构拥有我是否被允许尝试的最终决定权呢？
If I understand the risks, my doctor understands the risks, and there's real evidence a treatment could help, why does a regulator in Washington get the final word on whether I'm allowed to try?
当特朗普总统在2018年签署《患者尝试治疗权利法案》（Right to Try Act）时，国会就已经认可了这一原则的一部分。该法案是一个重要的进步，因为它明确指出：对于那些患有危及生命的疾病的患者来说，当所有已获批准的治疗方案都已用尽，且医生认为某种实验性治疗方法可能有效时，患者不应被当作自己生存过程的被动观察者。
Congress already recognized part of this principle when President Trump signed the Right to Try Act into law in 2018. That law was a major step forward because it affirmed something patients facing life-threatening diseases have always known: when approved options are exhausted, and a doctor believes an investigational treatment may help, patients should not be treated as passive observers in their own survival.
《患者尝试治疗权利法案》的出台并不意味着这一理念的终结，而是一个以患者为中心的医疗理念的开始——这一理念应该更广泛地影响美国食品药品监督管理局（FDA）的运作方式。在FDA评估罕见病患者的病情、风险、治疗紧迫性以及治疗的可及性时，同样应该遵循这一原则。如果患者和医生都清楚治疗的风险，并且有确凿的证据表明这种治疗方法可能有效，那么医疗体系应该选择与患者合作，而不是采取家长式的管理方式。
Right to Try was not the end of that idea. It was the beginning of a patient-centered philosophy that should shape the FDA’s culture more broadly. The same principle should apply when the agency weighs evidence, risk, urgency and access for rare-disease patients. If the patient understands the risk, the physician understands the risk, and there is real evidence that a treatment could help, the system should lean toward partnership instead of paternalism.
正因如此，参议员罗恩·约翰逊（Ron Johnson）和众议员戴安娜·哈什巴格尔（Diana Harshbarger）提出了“患者尝试治疗权利2.0”（Right to Try 2.0）法案，旨在将这一理念带入医学的新时代，特别是那些无法被纳入FDA传统审批流程的个性化治疗方案。他们推动这一改革的做法是正确的。不过，FDA的下任领导不应再等待国会的强制干预，而应该主动采取更加以患者为中心的治理方式。
That’s why Senator Ron Johnson and Representative Diana Harshbarger introduced Right to Try 2.0: to carry that same principle into the next era of medicine, including individualized treatments that do not fit neatly into the FDA’s traditional approval model. They are right to press the issue. But the next leader at the FDA should not have to wait for Congress to force a more patient-centered approach.
FDA早已意识到时间的重要性：其加速审批程序的存在正是为了防止那些患有严重、危及生命的疾病的患者因漫长的等待而失去宝贵的治疗机会。事实上，FDA已经通过这一程序批准了多种用于治疗杜氏肌营养不良症（DMD）的基因疗法。
The FDA already knows that time matters. Its accelerated approval pathway exists precisely because making patients with serious, life-threatening diseases wait years for a traditional clinical endpoint can cost them the thing they can't get back.
史上首个用于治疗听力损失的基因疗法获得FDA批准：这一进展具有“突破性意义”——因为对于患有杜氏肌营养不良症的患者来说，等待时间的长短直接关系到他们的健康状况。每一年的等待都可能意味着他们体力的进一步衰退、生活能力的进一步受限，以及独立性的进一步丧失。
The agency has used that pathway for multiple DMD treatments already. FIRST-EVER GENE THERAPY FOR HEARING LOSS GETS FDA APPROVAL: 'GROUNDBREAKING' Because waiting is never neutral when you have Duchenne. Every year can mean another loss of strength, another limitation, another piece of independence gone for good.
我的兄弟也患有DMD，他同样见证了这场战斗的另一面。即使治疗方法存在，真正获得治疗本身也是一场战斗。在华盛顿的会议室里，为了多年的临床试验长途跋涉听起来似乎可行。但当你是那个在身体与你作对时还要踏上旅程的人，而不是能在自己的医院就能接受治疗时，情况截然不同。
My brother, who also lives with DMD, has seen the other side of this fight, too. Even when a treatment exists, actually getting it can be its own battle. Traveling hours away from home for a multiyear clinical trial sounds manageable in a Washington conference room. It looks very different when you're the one making that trip while your body is working against you as opposed to having access to the therapy at your own hospital.
癌症治疗延误在美国激增，医生警告体系“需要立即修复” 这就是为什么我希望下一任FDA局长能倾听像我这样的患者的声音。我们不仅仅是在书本上读到这种疾病，或在显微镜下观察它——我们每天都在与它共存。它在我们体内——编织在我们的DNA里。与杜氏肌营养不良共存迫使我比预期了解更多关于药物开发和监管途径的知识。
CANCER TREATMENT DELAYS SURGE ACROSS US AS DOCTOR WARNS SYSTEM ‘NEEDS FIXING IMMEDIATELY’ That's why I want the next FDA commissioner to listen to patients like me. We don’t just read about the disease in books or look at it through a microscope — we live with it every day. It’s in us — woven into our DNA. Living with Duchenne has forced me to learn more about drug development and regulatory pathways than I ever expected to know.
我希望FDA能与患者、医生和罕见病专家合作。这也是为什么我要说一些会引起争议的话：我希望无论谁领导FDA，都能任命Houman Hemmati博士为副局长。他的职业生涯在生物技术领域，而非政治圈，致力于为罕见病和退行性疾病构建治疗方案，而非仅仅从外部进行监管。他也是我的朋友。我知道这句话会招致什么。批评者会说我只是想让认识的人进屋子。他们说得对，我认识他。但他们忽略的是：他不仅了解我的病，他还与我一起经历过它。
I want an FDA that works with patients, physicians and rare-disease experts. That's also why I'm going to say something people will have opinions about: I hope whoever leads the FDA brings on Dr. Houman Hemmati as deputy commissioner. He has spent his career in biotech, not politics, building treatments for rare and degenerative diseases instead of just regulating them from the outside. He's also my friend. I know what that sentence invites. Critics will say I'm just trying to get someone I know into the room. They're right that I know him. What they're missing is: he doesn't just know my disease, he's lived it with me.
我不奢求谁承诺给我永远。我只是请求一个为我仍能看到的未来而战的机会。我已经活过了许多人预期的寿命。我还没准备好停下脚步。
I'm not asking anyone to promise me forever. I'm asking for the chance to fight for the future I still see. I've already outlived what a lot of people expected. I'm not ready to stop.
正在考虑Overton博士提名的参议员们也面临选择：确认一位将患者视为生存伙伴的局长，还是维持一个把我们当作旁观者的体系。时候到了。我也希望华盛顿终于能明白，我的时间值多少。
Senators considering Dr. Overton's nomination have a choice to make too: confirm a commissioner who will treat patients as partners in their own survival, or keep a system that treats us as bystanders to it. It's time. And I'd like Washington to finally understand what mine is worth.