Patients and Families

At SOM Biotech, we stand with patients and families who face the challenges of a rare disease diagnosis with limited treatment options. Neurological and rare diseases touch every part of life—physical, emotional, and social. Our mission is to transform CNS drug discovery by uniting AI-powered drug discovery with deep clinical expertise, thereby advancing therapies that make a real and lasting difference for patients and their loved ones.
Patients and Families
Below you’ll find information about the disorders we are focused on and how we are committed to supporting the communities they impact. 

Huntington’s Disease (HD)

Huntington’s disease is a rare, inherited neurodegenerative disorder that affects movement, cognition, and behavior. Symptoms progress over time and can include challenges with coordination, balance, fine motor skills, speech, and swallowing, as well as difficulties with planning, decision-making, memory, and maintaining focus. Emotional and behavioral changes are also common, such as depression, anxiety, irritability, apathy, sleep disturbances, and repetitive or compulsive behaviors.

While there is currently no cure for HD, there is a growing need for therapies that address the full spectrum of symptoms across all stages of the disease. SOM Biotech is working to develop treatments that manage the broader behavioral, cognitive, and psychiatric needs of people living with HD in a highly tolerable way.

Tardive Dyskinesia (TD)

Tardive Dyskinesia is a movement disorder caused by long-term use of certain medications, particularly antipsychotics or drugs that block dopamine receptors. TD is characterized by involuntary, repetitive movements—often of the mouth, tongue, face, trunk, or limbs.

These movements can be socially isolating, uncomfortable, and emotionally distressing. TD can appear even after medications are reduced or discontinued, making early detection and management essential.

SOM Biotech is exploring new therapeutic approaches that may offer meaningful symptom control for individuals affected by TD and improve their overall quality of life.

Tourette Syndrome

Tourette Syndrome is a neurological condition characterized by motor and vocal tics. These tics can range from simple movements (such as blinking or throat clearing) to more complex or disruptive behaviors. Many individuals with Tourette’s also experience associated conditions, such as ADHD, OCD, anxiety, or learning challenges.

Tics often begin in childhood, and while some people see improvement with age, others continue to experience symptoms into adulthood. The impact can vary widely affecting school performance, social interactions, self-confidence, and emotional well-being.

SOM Biotech is developing new therapeutic candidates that aim to address Tourette Syndrome with precision, supporting both symptom reduction and long-term quality of life.

Phenylketonuria (PKU)

Phenylketonuria is a rare metabolic disorder caused by a deficiency in phenylalanine hydroxylase (PAH)—the enzyme responsible for breaking down the amino acid phenylalanine. When phenylalanine builds up in the body, it becomes toxic to the brain.

If untreated, PKU can lead to intellectual disability, seizures, behavioral problems, and psychiatric symptoms. Early diagnosis through newborn screening and lifelong management is critical. Dietary restriction of phenylalanine remains the primary treatment, but new targeted therapies are urgently needed.


SOM Biotech is advancing small-molecule approaches designed to enhance PAH stability and function, with the goal of giving individuals with PKU more therapeutic options and improved daily living.

Our Commitment to Patient Communities

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Listening to patients and caregivers

We actively seek insights from patient communities to ensure our research and development priorities reflect real-world needs, challenges, and aspirations.

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Advancing innovative therapies

We focus on diseases with significant unmet medical needs—particularly in the fields of rare CNS disorders—where our AI-powered discovery platform can make the greatest impact.

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Collaborating with advocacy groups and clinicians 

Partnerships with patient organizations, foundations, clinical experts, and academic centers help us accelerate progress and improve outcomes.

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Supporting families throughout the journey 

We are dedicated to creating resources that empower patients and caregivers with knowledge, clarity, and compassion as they navigate complex disorders.