Fragile X Awareness Day

 

Fragile X Awareness Day

Children are gifts, and they are born with many opportunities. Whether they thrive or struggle in their lives can be influenced by their genes.

— TheKnowHow

Children: Gifts with Unique Opportunities and Challenges

Children are gifts, each born with a world of opportunities. Whether they thrive or struggle in life can often be influenced by their genetic makeup. Every child is unique: some navigate life effortlessly, others test our patience, and some have special needs, facing significant obstacles. Having a child with Fragile-X Syndrome can be challenging. However, understanding the condition and seeking early professional assistance can maximize their independence and help them thrive within their abilities.

Understanding Fragile-X Syndrome

Fragile X Syndrome (FXS) is the most common inherited cause of mild-to-severe intellectual disability. According to Orphanet, a non-profit database for genetic diseases, approximately 1 in 2,400 children is born with Fragile-X. Infants show no symptoms at birth, but around one-third of affected children exhibit features of autism and delayed speech during early childhood. Hyperactivity and seizures may also be present. Physical characteristics, often subtle and easily overlooked, may include a long, narrow face, a prominent jaw and forehead, hyperflexible fingers, and large ears. After puberty, males may have larger testicles.

The FMR1 gene on the X chromosome causes Fragile-X Syndrome. The term “Fragile X” comes from the appearance of the X chromosome in affected individuals, which seems to be fragmented or broken. Human cells typically have 22 pairs of chromosomes. Females have two X chromosomes, while males have one X and one Y chromosome.

The FMR1 gene can be modified in three ways: “intermediate,” “premutation,” and “full mutation.” A normal FMR1 gene produces the protein FMRP (Fragile X Mental Retardation Protein), essential for brain development. People with a full mutation cannot produce this protein, resulting in Fragile-X Syndrome. Though primarily seen as a brain condition, FXS also affects other bodily processes. Recent research indicates that individuals with a less severe genetic change, known as “premutation,” may experience various medical issues, including fragile X-associated primary ovarian insufficiency (FXPOI), which impairs female fertility, and fragile X-associated tremor/ataxia syndrome (FXTAS), characterized by tremors and gait disorders.

Signs and Symptoms of Fragile-X

Children affected by FXS may show

  • developmental delays (not sitting, walking, or talking at the same time as other children the same age)
  • learning disabilities (trouble learning new skills)
  • social and behavior problems (such as not making eye contact, anxiety, trouble paying attention, hand flapping, acting and speaking without thinking, and being very active).

Boys with FXS often have some level of intellectual impairment, which can range from mild to severe. Females with FXS may have normal intelligence or some level of intellectual handicap. Autism spectrum disorder (ASD) is also more prevalent in those with FXS.

How Fragile-X is Inherited?

Understanding how FXS is inherited requires knowledge of the FMR1 gene alterations that cause FXS and other fragile X-associated illnesses.

Simply said, genes are lengthy sequences of four separate chemical compounds denoted by the letters A, T, C, and G. The order in which these letters appear determines the kind of protein produced by this gene. The chemical letters CGG appear often in the FMR1 gene. Different persons have varying numbers of CGG repetitions, although the majority have less than 45. People with FXS almost invariably have more than 200 repetitions, which cause the FMR1 gene to “turn off.” A blood test conducted by a healthcare professional or genetic counselor can identify a person’s CGG repeat count.

Normal: 5 to 44 Repeats

A typical amount of CGG repeats in the FMR1 gene ranges from 5 to 44. People with a normal number of repetitions do not develop FXS and do not pass on an increased risk of FXS to their children.

Intermediate: 45 to 54 Repeats

People who have an intermediate number of repeats (45 to 54) do not have FXS and are not at risk for having children with FXS. However, they may have a slightly higher chance of having some symptoms related to other fragile X-associated disorders and may pass the slightly higher chance of having these disorders to their children.

Premutation: 55 to 200 Repeats

People with 55 to 200 repetitions are considered to have a “premutation” in the FMR1 gene. They do not have FXS, but they may have or acquire, other fragile X-associated conditions. 
 With each pregnancy, a woman with a premutation in one of her FMR1 genes has a 50% chance of passing on either the premutation or a full mutation to her child. A male with a premutation will pass it down the premutation to his daughters but not his sons.

Full Mutation (FXS): More than 200 Repeats

People with a full mutation (more than 200 repetitions) have FXS. With each pregnancy, mothers with a full mutation have a 50% risk of passing fragile X on to their child (son or daughter). A male with full mutation will pass on his mutation to his daughters but not his sons.

Fragile-X: Genetic Testing Recommended

When speech and developmental delays become apparent, parents may consult a pediatrician or a family doctor. However, research has shown that the diagnostic journey finally resulting in genetic testing for FXS in children takes 18 to 24 months after parents voice concerns. This delay causes unnecessary stress on families and gaps in offering services for the kid. In many situations, families have a second child with FXS before the first is diagnosed.

The identification of a child with FXS might trigger cascade testing across the family. It may lead to the discovery of other family members who carry the complete mutation or a premutation.

Treatment of Fragile-X

Currently, there is no cure for FXS. However, treatment programs can assist afflicted children in developing important skills. Therapy to improve speech or social interaction may be included in the services provided. The sooner fragile X is discovered, and therapy is provided to the child, the better the child and their family will cope with the disease and foster a supportive and positive environment.

To create the best treatment plan, children with FXS, parents, and healthcare providers ought to work together closely, as should everyone involved in treatment and support — which may include teachers, childcare providers, coaches, therapists, and other family members. Taking advantage of all available resources will aid in success.

Finding Support

Having support and community resources can help raise confidence in managing FXS, promote quality of life, and aid in addressing the needs of all family members. It may be helpful for parents of children with FXS to connect with one another. One parent may have discovered how to address some of the same worries another parent has. Parents of special-needs children may frequently provide recommendations on useful resources for them.

Remember that what works for one family may not work for another. It is important that parents understand all options and discuss them with their child’s healthcare providers.

Sometimes, living with children with special needs can be full of laughter. However, fragile-X situations vary and might be troublesome. Not every family has the resources to devote adequate time and emotional investment to the ongoing support of the afflicted kid — or several impacted children. As precious as each child is, genetic counseling and family planning should not be disregarded when a family would be overwhelmed by another child with fragile-X.

Your Family is Affected? Read More about Fragile-X!

You have a child with special needs? You are seeking for more information?

We have found High Hopes, a non-for-profit pediatric therapy center in Dubai. They believe in their “super special ones” and explain in detail, how kids with fragile-X can be supported. Have a look:

High Hopes Dubai: Fragile-X syndrome

You have been diagnosed with a premutation?

We found information from Emory University School of Medicine Department of Human Genetics for you. Have a look:

Women´s Health and the Fragile X Premutation

Men’s Health and the Fragile X Premutation

Talk to your doctors, get support! Your Health matters!

TheKnowHow — Your Health, Your Voice
https://theknowhow.ae/blog/


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