About Fight CMD
Our Mission
We are dedicated to pioneering genetic breakthroughs for children with Congenital Muscular Dystrophy. Through powerful collaborations between scientists, clinicians and supporters, we are delivering hope and new therapeutic possibilities to families facing a lack of alternatives.
Betsy's Story & Our Foundation
Our two-year-old daughter, Betsy, was diagnosed with an extremely rare genetic neuromuscular disorder called Congenital Muscular Dystrophy (CMD). Her specific disorder is considered a dystroglycanopathy, which affects many areas of her body, including her muscles and brain. Unfortunately, time is not on Betsy’s side and things will get much worse as she gets older.
When Betsy was diagnosed, we were told that there is nothing that we can do but watch and wait. As her parents, we refused to sit around watching and waiting as our daughter regressed. Fight CMD was created to use advancements in science to develop a treatment option for children facing Congenital Muscular Dystrophy.
Our two-year-old daughter, Betsy, was diagnosed with an extremely rare genetic neuromuscular disorder called Congenital Muscular Dystrophy (CMD). Her specific disorder is considered a dystroglycanopathy, which affects many areas of her body, including her muscles and brain. Unfortunately, time is not on Betsy’s side and things will get much worse as she gets older. When Betsy was diagnosed, we were told that there is nothing that we can do but watch and wait. As her parents, we refused to sit around watching and waiting as our daughter regressed. Fight CMD was created to use advancements in science to develop a treatment option for children facing Congenital Muscular Dystrophy. While advancements in precision medicine and gene therapy offer a glimmer of hope for Betsy, the traditional treatment development pipeline remains a major obstacle. To give her a real chance, we must build an entirely new treatment customized to this type of CMD. We have partnered with top researchers to develop a therapy to slow the progression of Betsy’s disorder. Our mission is to fight for a cure so that children facing CMD have a treatment option and are given a chance at life. No family should have to watch and wait helplessly with no treatment options.
The Children We Fight For
Our drug development program is focused on gene therapy approaches to treat B3GALNT2-Related Congenital Muscular Dystrophy. Betsy and her friend, Hugo, are two of less than 10 children worldwide with this mutation. Our hope is our efforts to develop a treatment for this mutation will have broad implications for each child and family fighting CMD.
Betsy
Betsy was born a full-term, perfectly healthy baby girl. Although she had poor latch and didn’t cry for her feedings, we thought everything was normal.
At 4 months old, Betsy was not making her milestones and was diagnosed with severe hip dysplasia. She was casted and then braced, which further delayed her milestones. After this, we thought that she would finally be able to catch up, but she didn’t. At 12 months old, Betsy was still unable to sit up on her own, drooled excessively, had difficulty with eating and drinking, struggled with sleeping and was not talking, but communicated excitement with a raspberry sound that we lovingly call the “elephant sound.” At 18 months old, after continuing to be delayed despite early interventions, it was suggested that she have genetic testing.Betsy was diagnosed just before her second birthday with a rare genetic neuromuscular disorder called Congenital Muscular Dystrophy (CMD). Her specific disorder is considered a dystroglycanopathy caused by a mutation in the B3GALNT2 gene. This affects many areas of her body, including her muscles and brain. Unfortunately, time is not on Betsy’s side and things will get much worse as she gets older if we do not act now and fast. If we do not find a way to slow the progression of this disorder, Betsy could lose her ability to swallow, develop difficulty with breathing, develop heart issues, lose the ability to move and play, among other affects of this awful disorder. Despite the heartbreak that came with Betsy’s diagnosis, we decided to stay strong and fight for Betsy, our little elephant. It is our hope that we can use advancements in science and precision medicine to develop a customized treatment for Betsy and children like her who are facing the same type of Congenital Muscular Dystrophy. We dream about the things that Betsy will achieve in her life, with the help of the treatment that we develop.
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Hugo
Hugo, was born by cesarean section due to a breech position and the fact that he was unable to turn head down on time.
This caused his parents many concerns which was confirmed immediately after Hugo's birth: he didn't make any sounds, had no 'moro' reflex, he was sleepy and barely moved. His mother sensed something was wrong but after two days they were discharged and Hugo was labeled "lazy." At home she quickly noticed muscle spasms, which subsided after six months of his life. Hugo couldn't reach for toys, roll over, hold his head up. He had poor latch, developed significant sleep and bowel movement problems and didn't reach any milestones on time. He was taken to a hospital in Poland where they were immediately told that they'll be dealing with an ultra-rare muscle disorder, which was ultimately confirmed by a genetic report that came back to them when Hugo was one year old. Somehow, they didn't give up and through hard, constant work, they taught Hugo to sit and walk with assistance. They use all the therapies suggested by physiotherapists, such as Bobath, Medek, Vojta, massage, work on reflexes, balance, salivation, etc. Today, Hugo is 5 years old, but he still doesn't crawl, doesn't speak, drools excessively and requires assistance with everything he does (eating, toileting, bathing, getting up, walking, etc.). He also has a specific sleep disorder that significantly impacts his quality of life, as well as ours. His parents know that without appropriate treatment they will encounter insurmountable barriers. They dream that one day Hugo will achieve much more and that they will be able to cure this insidious disease that slowly robs them of everything we work for every day.
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Mara
Mara was born in August 2021. The birth was spontaneous and without complications. She was very small and tiny from the beginning, but otherwise unconcerned.
Unfortunately, I could only breastfeed her for three months, because she never really got full and additionally needed the bottle. However, swallowing and eating did not provide her with any problems.
Nevertheless, we noticed early on that something wasn't right. Mara developed significantly slower in all areas than other children. At one year old she could neither crawl nor walk. At the age of 16 months, we finally started with a comprehensive diagnostic. The pediatrician drew blood and a short time later we got the call that her creatin kinase and her liver were elevated.
After that we were transferred to human genetics. There it was discovered that Mara was carrying a change in the DMD gene (Duchenne muscular dystrophy). However, we could not accept this diagnosis at first, because it was explained to us that usually only young people develop symptoms.
That's why we turned to the Hamburg Uniklinik. Exome sequencing was done there. Shortly after, we received the final diagnosis: a CDG syndrome with an extremely rare mutation in the B3GALNT2 gene. Education was difficult, as there are very little information and experts on this rare disease.
To this day, Mara drools very strongly and has distinct problems sleeping. She's having a hard time sleeping through her first year of life. She is awake for several hours almost every night. She is significantly impaired in her linguistic and cognitive development. Her development does not correspond to her age, and unfortunately, she cannot speak so far.
Since May 2025, we will be a member of the GlykoKids association - a network for affected children and their families. The exchange and being with each other gives us a lot of strength. Nevertheless, we have not yet met a family whose child has the same mutation as our Mara. So far in Germany it is a unique case. Mara is an incredibly friendly, cheerful girl, who almost always laughs and has a very special sense of humor.
We will never give up hope that one day our little angel can be helped🙏🏻
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Our Board Members
Tyler Harris
Tyler Harris is founder, President, and CEO of Fight CMD.
He is a Principal Financial Analyst at Raytheon (RTX) supporting the Naval Power Division. He has over 9 years of experience in the defense industry. Prior to Raytheon, he worked in Healthcare Finance for nearly 7 years with Ochsner’s Health System, Children’s Hospital of New Orleans, Singing River Health, and other institutions.
He holds an MBA and a BS in Banking & Finance from Mississippi State University. He also completed an additional BS in Accounting from the University of Southern Mississippi.
Amanda Harris
Amanda Harris is the co-founder and vice president of Fight CMD.
Amanda Harris is the co-founder and vice president of Fight CMD. She practices medicine as a board-certified Physician Assistant in dermatology. She holds a BS in Biological Sciences from Louisiana State University and a Masters of Physician Assistant Studies from LSU Health Sciences Center in New Orleans. As an undergraduate student, Amanda was involved in a research study aimed at identifying an environmentally friendly termiticide, which was published in the Journal of Economic Entomology. She combines her medical and research background with a personal mission: to create a groundbreaking treatment for CMD to help her daughter, Betsy, and other children like her.
Brooke Abraham
Brooke Abraham is the secretary of Fight CMD. She works in HR in talent acquisition.
Brooke Abraham is the secretary of Fight CMD. She works in HR in talent acquisition. She studied General Studies at Louisiana State University. She is passionate about supporting the treatment development for CMD for her niece, Betsy, and other children like her.
Our Core Values
Hope
We believe a treatment for CMD is possible and within reach
Innovation
When fighting a progressive disease, each new discovery matters.
Community
Together, we are stronger and more effective
Support our work
Fighting Congenital Muscular Dystrophy
FightCMD, a non-profit organization (Tax ID number 41-2756694), is qualified as a tax-exempt organization under section 501(c)(3) of the IRS and has been designated as a “public charity” under section 170.