Published on: Wednesday, August 7, 2024

Hope for Angelman Syndrome Patients: ION582 Eases Symptoms

Angelman syndrome, a rare genetic disorder, has been receiving more attention in recent years thanks to the efforts of celebrities like Colin Farrell, who launched a foundation to support adults with intellectual disabilities. Now, a new development in treatment brings hope to those affected by the condition.

What is Angelman Syndrome?

Angelman syndrome is a complex disorder that shares similar symptoms with cerebral palsy and autism. It affects approximately 1 in 15,000 people worldwide, causing developmental delay, intellectual disability, and speech impairment. Despite its prevalence, there is still a lack of understanding about the condition, making it challenging to diagnose and treat.

Trial Data Reveals Promising Results

In a recent Phase 1/2 trial, the treatment ION582 was found to be well-tolerated and effective in improving patients' symptoms. This breakthrough offers new hope for those living with Angelman syndrome, who often face a low life expectancy. The trial data is a significant step forward in the development of therapeutics for this rare condition.

As researchers continue to explore new treatment options, the awareness and support generated by initiatives like Colin Farrell's foundation are crucial in improving the diagnosis and care of Angelman syndrome patients.

Stay informed about the latest developments in Angelman syndrome research and treatment options. Learn more about this rare condition, its symptoms, and how you can make a difference.

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Angelman syndrome treatment ION582 eases symptoms: Trial data

Angelman syndrome treatment ION582 was well tolerated and was found to improve patients' symptoms, Phase 1/2 trial data showed.

What is Angelman syndrome? Colin Farrell launches foundation in ...

Foundation will provide support for adults with intellectual disabilities · Angelman syndrome has similar symptoms to cerebral palsy, autism · Farrell hopes to ...

Angelman syndrome - Symptoms and causes - Mayo Clinic

Angelman syndrome is a genetic condition that causes developmental delays, mental disability, seizures and happy demeanor. Learn about the symptoms, causes, diagnosis and treatment options from Mayo Clinic experts.

Angelman syndrome - Wikipedia

Angelman syndrome (AS) is a genetic disorder that mainly affects the nervous system. [6] Symptoms include a small head and a specific facial appearance, severe intellectual disability, developmental disability, limited to no functional speech, balance and movement problems, seizures, and sleep problems. [6]

What is Angelman Syndrome

Angelman syndrome (AS) is a rare neuro-genetic disorder caused by a loss of function of the UBE3A gene. Learn about the symptoms, causes, types, prognosis, history and cure of AS from the ASF website.

Angelman Syndrome: What It Is, Symptoms & Treatment - Cleveland Clinic

Angelman syndrome is a rare neurodevelopmental condition that affects the nervous system and causes developmental delays, intellectual disabilities and seizures. Learn about the genetic mutation, diagnosis, management and outlook of this condition.

Angelman Syndrome - National Institute of Neurological Disorders and Stroke

Angelman syndrome is a genetic disorder that primarily affects the nervous system. The disorder is named after Dr. Harry Angelman who first reported the syndrome in 1965. Features that help define the disorder include: Often, there are also gastrointestinal, orthopedic, and eye problems. Hyperactivity and a short attention span are common.

Angelman syndrome - MedlinePlus

Angelman syndrome is a complex genetic disorder that primarily affects the nervous system. Characteristic features of this condition include delayed development, intellectual disability, severe speech impairment, and problems with movement and balance (ataxia). Most affected children also have recurrent seizures (epilepsy) and a small head size ...

Angelman Syndrome - StatPearls - NCBI Bookshelf

Angelman syndrome is a rare disorder caused by loss of function of the maternal UBE3A. It presents in childhood with psychomotor delay, absent speech, ataxia, and motor impairments. This activity reviews the evaluation and management of Angelman syndrome and the role of the interprofessional team in managing patients with this condition ...

Angelman Syndrome Foundation – With you for the journey

The mission of the Angelman Syndrome Foundation is to advance the awareness and treatment of Angelman syndrome through education and information, research, and support for individuals with Angelman syndrome, their families and other concerned parties. We exist to give all of them a reason to smile, with the ultimate goal of finding a cure.

Angelman syndrome - Diagnosis and treatment - Mayo Clinic

Diagnosis. Your child's healthcare professional may suspect Angelman syndrome if your child has developmental delays, talks little or not at all, or has other symptoms. Symptoms might include seizures, trouble with movement and balance or a small head size. Angelman syndrome can be hard to diagnose because it shares symptoms with other types of ...

Angelman syndrome - NHS

Angelman syndrome is a rare genetic condition that affects the nervous system and causes severe physical and learning disabilities. Learn about the characteristics, diagnosis, management and support for people with Angelman syndrome and their families.