"Together as One" means teamwork and collaboration, working together with a shared passion and determination to get our product to our patients as quickly and safely as possible.

I am inspired by--and try to emulate in my work--the determination, compassion and hope of the rare disease community.

As a #RareAlly, I work cross-functionally with my fellow Taysha team members to support our ongoing effort to eradicate monogenic CNS diseases. We are true allies to all of our stakeholders, including our patient community, by being transparent through proactive communication.

By day, I work collaboratively with my colleagues to make gene therapies for rare disorders a reality. On the weekends, my dog Watson and Cricket help me bring joy to kids with rare disorders at children's hospitals or the Ronald McDonald House.

#RareAlly means being a partner, ensuring that you listen, learn, and demonstrate collaboration in a meaningful way to the rare disease groups were are working with.

My mother and my wife have been my rock and inspiration. My mother as she is battling various rare illnesses that have altered her way of life, and she tries to set aside her health issues putting others before herself, which inspires me. My wife is a cosmetologist and at many occasions she would dedicate her talents making home visits to clients or others impacted by a rare disease in order to make them feel beautiful inside and out.

RareAlly is a very committed, diverse, and continually growing community. It's hyper-focused on addressing the needs of the community members impacted by an ever-increasing number of diseases that when characterized individually are considered rare.

The families who have loved ones that are living with a rare disease are my inspiration. I try to summon even a small percentage of their strength, perseverance and caring into how I interact in the world. The scientific progress in this area is due in large part to their efforts, and it inspires me to go further and work harder for them.

The opportunity to be a small part of the force for good

For me, "Together as One" means finding inspiration in Rare parents who love their children so deeply that they work on treatments, the Rare siblings who establish a new normal amidst an unfamiliar world and navigate adversity with poise and courage, and the Rare angels who teach so many of us to work harder and leave no stone unturned along the path to a treatment. It also includes the Rare researchers and Rare clinical development teams who work tirelessly with drive to discover novel therapies and provide hope for devastating diseases.

When I look at my son and think about the projects we are working on at Taysha, I can't help but feel the urgency to get our products in the clinic.

I am lucky enough to have two people close to me who are inspirations in rare disease. The first is my husband. In his professional life he is dedicated to treating children with rare disease and the ongoing work to identify ultra-rare diseases. The second person is my stepfather, who has a few personal links to rare disease. He decided to raise money for NORD through their Running for Rare team and complete a 'bucket list' goal by running his first marathon a few years ago at the age of 67. In addition to his training, he spent months focused on building awareness of rare disease through his personal networks. NORD paired him with a young woman living with Pompe disease and they developed a strong relationship in the months leading up to the race.

Being a #RareAlly is acknowledging the strength of individuals to inspire and impact change. Together, the possibilities are endless!

Supporting all those in the rare disease community.

Most rare diseases don't have a treatment, just because something is rare doesn't mean it is any less important. I'm proud to be working at a company trying to advance gene therapies to serve a population of patients who suffer from diseases with high unmet medical needs.

The founder of the Neuroendocrine Cancer Awareness Network. As an advocate and a patient, she and her husband Bob taught me what it means to be a part of the rare disease community. It has been over 10 years now and I am still in awe of the impact her and her team have on the neuroendocrine community through education, patient support, and research activities.

Every single patient

Six years ago I was diagnosed with Systemic Sclerosis (Scleroderma). I quickly felt a circle of love, strength and inspiration fold around me. This circle is comprised of my family, friends, co-workers and other sclero warriors living with this rare and presently incurable disease. As a Taysha employee, I feel honored to contribute one small piece towards improving patient's lives and ultimately eradicating monogenic CNS disease. I am deeply committed to being a part of our patients' circle.

All of us who are rare allies together are united by the common goal of helping those afflicted with severe and rare disease. We advocate for them, teach the world about rare disease on their behalf and do our best to understand the diseases from a scientific perspective so that we can discover and develop treatments that may be able to offer some help.

To me, #RareAlly means investing in and prioritizing those who are affected by some of the most uncommon and severe diagnoses. Rare Disease is particularly close to me as my grandfather fought a long, hard battle with ALS. I am proud of Taysha and all the hard work we are doing to advocate for these patients with the ambitious goal of eradicating monogenic CNS disease.

Anika is my #RareAlly inspiration. Her daughter, Leilani was born with 5p minus syndrome (AKA Cri-du-chat). Leilani was diagnosed as an infant and Anika has been an amazing advocate for her since day one. I'm in awe of how strong she has been through challenging times, including a major surgery required to correct Leilani's spine. Anika recently published a children's book about Leilani to share how special her daughter is with the world and raise awareness around rare disease. She is the epitome of being a #RareAlly.

Coming together as one I think takes on new meaning. Anytime I speak to a family that has a child with Rett syndrome, whether that child was diagnosed that day or whether that diagnosis came several decades ago, there's always an immediate bond. I've met an incredible group of people, parents like myself who have children with Rett syndrome, donors, philanthropists, scientists and patient advocates. Rett syndrome is a rare disease. It doesn't feel rare when you have a child with Rett, it doesn't feel rare to us. We may be rare, but we're really powerful when we come together to fight Rett syndrome.

Of course, I have to say it would be Amelie. For us, her memory will always live on in our hearts at the CATS Foundation. What she went through as a child really gave us the strength and determination to help others to make sure that they had a smoother journey through that entire process. She’s inspired us to set up a foundation that not only provides a community, but it provides people with that ability to find out the latest information. Our support network is now so strong that we know we have these people we can turn to help us through those difficult moments.

My rock in my #RareAlly is my daughter, Macy, who was diagnosed with Rett syndrome. Looking at her every day inspires me. I can't imagine someone stronger than a child who gets up every day and battles what she has to battle, and to do so with a smile on her face. She just gives me this great, humble perspective that keeps me grounded and keeps me going. How could I not fight for this child? I had a passion for others and giving to others. Little did I know that that passion was going to be funneled directly into Rett syndrome, which Macy brought into that right turn in life.

I understood the true measure of being an ally when my daughter’s 8th grade classmates organized a fundraiser for CLN1 disease research. Imagine a school gym filled to capacity with students, teachers and staff all dressed in pink and purple and dancing their hearts out. My blind daughter was in a circle with her closest girlfriends jumping up and down to the beat with a smile that told me everything I needed to know. I believe that day was one of the happiest of her life. It was also a life-changing day for the other participants. Every person in the gym was a #RareAlly united in support of one of their own. The kids raised a lot of money for research that day, but it paled in contrast to the love, care, and memories we will forever share.

As a #RareAlly, I strive to learn as much as I can from those living or caring for someone with a rare disease. They are the experts and by working together, we can make a difference.

I was deeply impacted by the story of a family who adopted a child with a rare disease, who had been born in a foreign country and abandoned in a ditch because of his disease. The love and dedication that the adoptive family has for this child is such an inspiration and the greatest example of what being an ally for these children means. I am also inspired by the families who have lost children to rare disease because many have continued supporting the cause by creating foundations and support groups to help other families who have been impacted by similar conditions as well.