Commercial drug development focuses on developing drugs that can serve large populations to be profitable (e.g., diabetes). The disease that William has impacts only an estimated 15,000 patients in the US and his subtype is even rarer. The drug we are working to advance into clinical trial was designed for this subtype of Duchenne and will begin clinical trials once manufacturing and toxicology is completed

Insurance providers typically do not cover treatments that are part of clinical trials. Clinical trials are considered experimental by nature, meaning the treatments being studied have not yet been proven effective through the full scientific process. Because of this, there is no established proof of concept, which is a core requirement for insurance coverage.

Additionally, insurance is not structured to fund research or experimental therapies. Instead, clinical trials are usually supported through other avenues such as government grants, academic institutions, pharmaceutical companies, or private foundations. This funding model helps ensure that the financial risk of unproven treatments does not fall on insurance providers until the therapies are validated and approved for widespread use. Unfortunately, given the rarity of this disease and its subtype, no commercial entity will ever be financially motivated to develop a drug without the potential for significant downstream profitability.

The total cost of this effort is allocated directly to the development and final safety testing of the treatment:

$1.6 million is the contract amount with the pharmaceutical company to manufacture the drug. This is structured as 50% due upfront to initiate production of the treatment (IN 2025), and 50% due upon completion (6 months later)

$600,000 is designated for the toxicology study, which will begin once the treatment is completed and handed over, this study takes 4 months.

None of the funds raised are being used for administrative costs or overhead. Every dollar is dedicated entirely to the direct development of this drug treatment.

This project has the potential to create a pathway not just for one child, but for the entire Duchenne community and other rare disease families. Success here would demonstrate a model for how targeted treatments can be developed faster and with greater efficacy and efficiency — offering hope for future therapies that may otherwise never be pursued due to limited commercial incentives. Every step forward in this process helps build infrastructure and proof points that can accelerate solutions for countless others.

CRD works hand in hand with us and their families to fundraise together. Moreover, CRD leads the drug development effort and ensures that the drug is developed to the highest quality possible. CRD itself does not have millions of dollars in its war chest waiting to deploy, and is continuously working with and alongside families to fundraise the capital needed to advance potentially life saving drugs into the clinic and ultimately delivered to patients who desperately need them.

At Cure Rare Disease, this work isn’t just professional—it’s deeply personal. Every member of the team has been impacted by rare disease. They’re parents, siblings, friends and patients themselves who’ve sat in hospital rooms, heard the words “no treatment,” and felt the crushing weight of helplessness. That pain drives their commitment to create a different future—not just for their own families, but for every family facing the same devastating reality.

They’ve built strong partnerships with leading researchers, manufacturers, and the FDA, because they know families shouldn’t have to figure this out on their own. They’ve walked that road, and no one should have to do it without support. Their role is to carry the burden of drug development so families can focus on loving their child—not chasing science, regulatory partners or approvals.

This mission is more than a job. It’s a promise born from their own heartbreak to fight for every life that others may overlook. And while actively pursuing large-scale funding avenues that often take far too long to access, it’s the support of family-led fundraising that allows CRD to move the needle forward now. That support is not only powerful… it’s life-saving.

They also have a dedicated fundraising professional who works hand in hand (day in and out) who helps families get campaigns off the ground. They are here because they know what’s at stake. And they will never stop pushing for answers, for hope, and for time.

We will not stop. Our commitment to developing this treatment is unwavering, and we will continue raising funds until the full amount is secured. If the goal isn’t met on the original timeline, it may shift back by a few weeks or months — but this is and will remain our #1 priority. The younger and healthier William is when the treatment is ready, the better positioned he will be to receive it. Our hope is to avoid any delays, and if any occur, they will be as short as possible. Encouragingly, we have already raised $300,000 in less than two weeks, and we are pushing forward every day.

No. Duchenne is a degenerative muscle wasting disease with no cure. Since Duchenne is a genetic abnormality, treatment of the condition is focusing on groundbreaking genetic and gene editing technologies.

Duchenne Muscular Dystrophy is a rare genetic disorder that affects around 1 in every 5,000 people. Mostly boys.

No, there are a variety of forms of muscular dystrophy, but Duchenne is the most common type. Duchenne occurs when there is a genetic abnormality on the gene that causes dystrophin production. This genetic abnormality can take a variety of forms: a deletion, or a duplication, and it can occur anywhere on that gene.

Dystrophin is a protein found in muscles that works to strengthen and repair muscle fibers after they contract and relax. Duchenne patients produce either small amounts of or no dystrophin at all, which renders them unable to replenish muscle strength as they grow and use their bodies.

William’s prospective treatment will use the gene-editing technology known as CRISPR to change the code written in William’s body. You may have seen headlines about this exciting development coming out of the science world. CRISPR-Cas9 is a unique technology that enables geneticists and medical researchers to edit parts of the genome by removing, adding or altering sections of the DNA sequence.

In 2025 we learned we have some actual CRISPR solutions for William’s exact mutation. FDA has given pre-approval, and we are heading to drug manufacturing by early fall 2025.

Of course, William’s progression, our fundraising efforts and family’s needs will all be part of this timeline.

Some of the first symptoms may include frequent falls, difficulty running or standing up, waddling gait, enlarged calves, and sometimes delayed developmental milestones. Eventually, continued muscle deterioration results in losing the ability to walk. In the later stages of the disease, respiratory insufficiency or cardiomyopathy are common and life-threatening.

Currently, William’s symptoms are showing. He can do activities similarly to many of his friends but typically slower and if you watch him, you will notice more tripping, falls and frustrations with gross motor skills. He does get tired after a busy day and we monitor his activity and make sure he’s rested and hydrated. Often, we will have to skip activities if they are too much or take rest days in between big activity days.

William is mostly asymptomatic thus far, so we don’t dwell on it with him at the moment. For now, we are making sure he is aware that his muscles work a little differently than most kids his age so he can advocate for more rest when needed.

DMD is rare so educating yourself is a good first step. Thank you for being here. Talking about William and DMD to your friends and family helps to spread awareness as well. On a very practical level, this medical treatment is extremely expensive and any donation to this cause is helpful. If you have children, we love the idea of expanding your library to include stories with children who use wheelchairs to help normalize wheelchairs for kids. Don’t forget to spread love and kindness, as life is short.

To an outsider, William leads a relatively typical life. However, although his symptoms are mild at this time, we have things to consider on a daily basis. Our family is aiming to reserve his muscle use, which means we do small things like lift him to the top of a slide instead of letting him run up the stairs. He also wears AFO’s (braces) on his legs at night to help stretch his Achilles tendons and support his calves. He already has more medical appointments than an average child in order to establish baselines and monitor his health.

We are still learning how this process will play out, but our guess is that the treatment will be an IV bag, in a hospital with close monitoring afterwards. It seems so basic, but that’s how science works.

We have asked this question of other DMD parents and of William himself. At this time, it doesn’t seem William is in any pain.

As of 2022, DMD is 100% fatal. It is a horrific prognosis. Historically, Duchenne patients can be expected to live into their early 20s, but thanks to advances in treatment are sometimes now living to their early 30s and beyond.

Duchenne affects mostly boys, and they will begin to see symptoms (typically) around age 3-5. It is a very unusual that we know this information without symptoms and without an older sibling with DMD.

Duchenne is a genetic disorder, so it is either passed down from parents or is a random birth defect. Kati has been tested and is not a carrier, so in William’s case it’s simply random.

CRISPRs are specialized stretches of DNA, and the protein Cas9 is an enzyme that acts like a pair of scissors, capable of cutting strands of DNA. The goal is that CRISPR technology can be used to correct gene duplications that cause DMD by restoring full-length dystrophin, a protein critical to muscle strength and function.