Living with ahds
At Alera Bio, we understand the daily impact of MCT-8 deficiency, not only on the patients but also on their families and caregivers. Through our commitment to advancing new treatments, we aim to improve the lives of individuals affected by this condition and offer hope for a better future.
Alera Bio:
A Future of Hope and Possibility
Living with AHDS
MCT-8 deficiency, also known as Allan-Herndon-Dudley syndrome, is a rare genetic disorder that primarily affects the nervous system, leading to severe developmental and motor impairments. While each patient’s experience with MCT-8 deficiency is unique, the challenges are often profound-affecting mobility, cognitive function, and overall quality of life.
Symptoms and Impact
MCT-8 deficiency results from mutations in the gene responsible for the transport of thyroid hormones to the brain, leading to neurological deficits. The severity and progression of symptoms vary, but common challenges include:
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Severe Motor Impairments: Most patients experience significant delays in motor skills, including problems with movement, coordination, and muscle weakness. This can lead to the need for mobility aids, such as wheelchairs or braces, and result in limited independence.
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Cognitive and Developmental Delays: Children with MCT-8 deficiency often experience delays in speech, language, and overall cognitive development. Intellectual disabilities may range from mild to severe.
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Neurological Symptoms: Seizures, muscle rigidity, and difficulty swallowing are common symptoms that further complicate daily life.
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Shortened Lifespan: Due to complications such as respiratory distress and other life-threatening issues, MCT-8 deficiency may lead to a reduced life expectancy.
Emotional & Social Impact
Living with MCT-8 deficiency goes beyond the physical challenges; it deeply affects the emotional and social lives of patients and their families. The emotional burden of caring for a loved one with this rare condition can be overwhelming. Families often face:
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Caregiver Stress: Family members often take on the role of primary caregivers, providing 24/7 care for their loved ones. This can result in significant emotional and physical stress.
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Social Isolation: The rarity of the disease often makes it difficult for families to find local support groups, leading to feelings of isolation.
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Educational Challenges: Children with MCT-8 deficiency may require specialized educational programs to support their development. This may place additional strain on families as they navigate the educational system to ensure the best care and opportunities for their children.
A DEVASTATING CONDITION FOR CHILDREN AND THEIR FAMILIES
Our Commitment to Change
Serious Conditions warrant Serious Innovation
(that's what motivates us each day!)
At Alera Bio, we believe that no one should have to face MCT-8 deficiency without hope. We are dedicated to advancing treatments for this rare condition and improving the lives of those affected. Through innovative research and clinical trials, we aim to provide new therapies that address the underlying causes of MCT-8 deficiency, giving patients and families the opportunity for a better quality of life.
While there is no cure for MCT-8 deficiency yet, we are committed to making progress. Our investigational treatment, currently in preclinical development, is designed to restore thyroid hormone transport in the brain, potentially improving motor function, cognitive abilities, and overall health.
We envision a future where children with MCT-8 deficiency can thrive – where they can live more independent, fulfilling lives. This vision drives everything we do at Alera Bio. We are committed to pushing the boundaries of science to bring about real, tangible change for those affected by this condition.
Join Us on Our Journey
We understand the importance of support and inclusion in the fight against MCT-8 deficiency. As we prepare to enter clinical trials in 2025, we invite families, caregivers, and healthcare providers to join us on this journey. Together, we can make a meaningful difference in the lives of patients living with MCT-8 deficiency.
For more information on how to get involved, to share your story, or to learn more about MCT-8 deficiency and our research, please contact us or sign up for updates.