Crowdfunding campaign to raise awareness about Cystinosis in children

Have you heard about the disease Cystinosis? Did you know that it occurs mainly in children? The disease Cystinosis is hereditary, autosomal recessive and does not affect all generations. Only some children are susceptible to it. This affliction causes deposition of the amino acid cysteine in the cells, leading to tissue damage. Amino acids are the main components of proteins, and cysteine is partly synthesized by the body and partly absorbed from food.

Cystinosis in children affects a wide range of newborns worldwide and statistics show that it occurs in 1 in 100,000-200,000. Despite these figures there are places in the world where this disease has a higher incidence. This is the case in the Brittany region of France. It has a higher incidence of around 1 in 26 000 births.

Today, with the Pavel Andreev Foundation, we're going to tell you a little bit more about this, and why it's important to raise awareness about Cystinosis in children. We'll also talk about how anyone can start their own charity campaign to raise awareness about Cystinosis. See in the coming lines:

Contents:

1) What is Cystinosis in children;

2) Treatment and prevention of Cystinosis in children;

3) Why it is important to raise awareness about Cystinosis in children;

4) How a Crowdfunding campaign to raise awareness about Cystinosis in children can benefit us.

1) What is Cystinosis in children

There are two main forms of the disease cystinosis. They are as follows:

• Nephropathic form

It can occur in early childhood, it is also called the classic form, which is also expressed in a more severe form. It is also present at a later stage when it manifests in early adolescence and is known as the intermediate form.

• Nephropathic form

This form of the disease is manifested by ocular symptoms. The nephropathic form is the most common manifestation of the disease and can be said to be more serious. It causes a specific type of damage in the kidney known as Fanconi syndrome. The disease proceeds in the loss of many important substances that are excreted, through the urine (water, electrolytes and minerals). To the manifestations of cystinosis of this type let us add:

Acidosis;

Frequent urination;

Photophobia and visual impairment;

Dehydration;

Growth retardation.

The platform at PavelAndreev.ORG is a charitable foundation whose goal and mission is to inform people and provide convenient navigation to start charitable campaigns. It is where people share their noble causes, tell about difficulties, problems and illnesses they or their loved ones have faced and where they find donors who help.

Users and donors of different social status, abilities and causes meet in this place, but they all have the ability to unite for the sake of good and noble deeds. Here at Pavel Andreev Foundation everyone can learn something new, do a good deed by becoming a donor or start a fundraising campaign for someone in need.

By choosing a proven international platform to organize a fundraising campaign you will add credibility and legitimacy to your cause and this will help you attract more people willing to donate as well as inform more users. See the next section for how cystinosis treatment and prevention works in children:

2) Treatment and prevention of Cystinosis in children

Cystinosis in children is an inherited disease in which cystine accumulates intracellularly and causes tissue destruction. Among the organs most affected are the kidneys and eyes. In these, severe long-term sequelae remain. The main treatment for this disease is by reducing the amounts of cystine from the cells. Early diagnosis and treatment provide a much better prognosis for cure and return of children to a normal lifestyle.

• Preventing/delaying the initial manifestations

Early treatment with drug therapies helps to reduce intracellular cystine content. This limits the extent of the disease. The drugs that are prescribed act on the principle of penetration into the cell and lysosome, forming complexes with cysteine and cystine. Such allow egress into the cytoplasm. Early measures, prevention and treatment can delay kidney disease. This sometimes even eliminates the need for a kidney transplant. Positive effects can also be achieved in the areas of thyroid dysfunction, muscle mass and growth rate. The effects of these are less pronounced when the medication is later administered because the effects already established are irreversible (an example of this is Fanconi syndrome). It is worth noting here that treatment with various forms of cysteamine is recommended at any age, preventing or mitigating the side effects of cystinosis. Very often the medications for such treatment are too expensive and it is not in everyone's power to be able to afford them. That's when fundraising campaigns come to the rescue to raise money to treat cystinosis in children.

• Symptomatic treatment

The most important manifestations that necessitate treatment of cystinosis in children are those conditioned by renal dysfunction. In this type of treatment and without taking into account the time of presentation of the disease, a substitute for renal losses is recommended. This is done by adequate measures, appropriate therapies and depending on the stage and losses. All of this is tailored by professional medical teams that care for patients. To help and support, anyone who wishes could start organizing a fundraising campaign.

Kidney transplant

A kidney transplant is a very effective way to combat this chronic kidney disease. There is a proven effective approach. However, in these cases it requires a lot of professionalism, medical teams, appropriate equipment and aftercare. Here, again, things come down to huge financial resources, which have to be provided one way or another. Fundraising campaigns help to raise these.

Why kidney transplantation is important

A kidney transplant dramatically changes the course of a disease that can be fatal around the age of 10. The pathogenic cysteamine treatment, first introduced in the 1980s, increases the life expectancy of these patients. Men with cystinosis remain infertile, although the potential effects of early and prolonged cysteamine treatment are unknown. There have been cases of women who have given birth to healthy children.

3) Why it is important to raise awareness about Cystinosis in children

Cystinosis in children is a rare disease and as a result of it not being a typically occurring problem carries great risks of reaching serious stages of development. It is important that awareness of cystinosis in children is raised so that people know how they can protect their future generation. When the risks are known and if there is a history of genetic predisposition in a family, prenatal diagnosis may be recommended. Such is administered in the first trimester of pregnancy. For this purpose, cells collected by chorionic villus biopsy or amniocentesis are examined. The level of cystine in amniotic fluid fibroblasts is assessed directly. Sometimes detection of the disease can also occur after genetic tests are done. This is how the presence of this disease can be diagnosed.

• It is important to raise awareness of this disease in order to anticipate and plan timely measures

One of the most important reasons for raising awareness about Cystinosis in children is that early detection and diagnosis can lead to successful treatment and improved lives.

Kidney transplantation is one of the treatment options for Cystinosis and can dramatically change the course of what has been achieved. This procedure increases the expected life change for patients and enables them to lead a more normal life.

Along with this, the use of cysteamine is also an important aspect of caring for children with Cystinosis. This drug, first introduced in the 1980s, has had a significant positive effect on changing patients' lives. However, awareness of the risks, symptoms how the disease progresses, treatment methods, and who may be affected is still low.

One reason why it is important to raise awareness about Cystinosis in children is the fact that men with this disease remain infertile. Although the potential effects of early and prolonged treatment with cysteamine are unknown, there have been cases of women who have given birth to healthy children. This shows that with proper treatment and care, should not be an obstacle to family happiness.

Raising awareness of Cystinosis in children can happen, with the help of organising a charity fundraising campaign.

Awareness of the problem can be essential because earlier detection of appropriate and timely treatment can significantly improve the prognosis and quality of life for patients. Dissemination of information about Cystinosis to parents, medical professionals and the community at large can contribute to early diagnosis and appropriate treatment of children suffering from this disease.

4) How a Crowdfunding campaign to raise awareness about Cystinosis in children can benefit us

Crowdfunding campaigns are transforming into an effective way to raise funds and awareness for various causes and diseases. In this case, a crowdfunding campaign to raise awareness for Cystinosis in Children can have significant benefits.

Start a campaign now on PavelAndreev.ORG.

As we have already mentioned, Cystinosis in children is a rare genetic affliction that affects the very young, even newborns. Due to its rarity, awareness of it is limited, as is funding for research and development of the treatment process. A charity fundraising campaign can help in this regard by raising funds from the public and raising awareness of Cystinosis.

The advantages of a fundraising campaign to raise awareness are many:

It can get the attention of people who have never heard of Cystinosis but are willing to help. An awareness campaign at the Pavel Andreev Foundation can create a broad community of supporters to share the information and spread it further. All this will work in a big plus for more awareness to the masses of people and also to support various research studies and discover and create better technologies and medical equipment to diagnose and treat the disease.

You can read more about Pavel Andreev Foundation posts on social media, where you will see for yourself that this type of charity helps not only people in need, but also anyone inclined towards charitable causes.

The funds raised, through the fundraising campaign, can be used for research that will help to better understand the kidney disease Cystinosis and implement more effective treatment methods. Funds can also be used to support the families of the children, providing them with information, support and resources.

Raising awareness of Cystinosis through a crowdfunding campaign can lead to earlier detection of the problem, which is essential for successful treatment and improved prognosis for unborn children. In addition, greater awareness can lead to better understanding and acceptance of the problem by the public.

May all these arguments be of benefit to anyone reading this thread at this time, and be a good starting point for anyone inclined to plan a fundraising campaign on PavelAndreev.ORG.