Our collaborations with patient organizations
Working together with Global and European patient organizations
In rare diseases we are working together with a number of Global and European patient communities.
‘Patient Engagement has been and continues to be at the core of Ipsen’s activities and we at WAPO are very appreciative of the continuous synergy we can offer to both industry and the global pituitary patient community.’
World Alliance of Pituitary Organizations (WAPO)
Uniting on health awareness
We know how important driving health awareness is, be that by dedicated months, weeks, days, or longer-term programs. Health awareness events are an important time when communities come together to raise awareness and show support for people living with rare conditions, their families, friends, and caregivers.
Rare Disease Day, 28 February
In 2021, for Rare Disease Day, Ipsen created The World’s Rarest Dictionary Project. The idea was for people in the rare disease community to create and submit entirely new words to describe what life is like living with, and caring for people with, rare diseases. The goal of The World’s Rarest Dictionary Project was to create a more relevant and relatable words used to talk about rare diseases.
Find out more here.
Projects developed together with patient communities
Together with patient communities around the world, we have worked on projects covering research and development, clinical study information, and patient support programs. The aim of these projects is to help make a difference for patients, their families, and caregivers.
Explore some our projects below:
Patient voice program in FOP
Ipsen in Spain collaborated with the Spanish patient association, Asociciacion Espanola de Fibrodisplasia Osificante Progresiva (AEFOP), to understand experiences of living with FOP in Spain, with the aim of helping to raise awareness of the condition and identifying where people living with FOP need more support.
Our areas of expertise
Rare diseases are defined as those that affect a small percentage of the population – for example, in Europe this applies to conditions that affect fewer than 1 in 2000 people, and in the United States as affecting fewer than 200,000 people.20,21 80% of rare diseases are genetic, which means they are caused by a change to a person’s DNA, this change can be spontaneous or passed down from biological parents. It is present throughout life and in some cases rare diseases are life-threatening.20
There are currently between 6,000 and 8,000 known rare diseases which affect around 300 million people around the world.22
In the Useful resources section of this page you will find links to Ipsen websites where you might be able to find additional information and support about different rare diseases.
You can also listen to stories from people affected by rare diseases in the Experiences of living with rare diseases section below.
Acromegaly is a rare type of endocrine, or hormonal, disorder where the body produces too much growth hormone, causing body tissues and bones to grow more quickly. This is most often caused by a benign (non-cancerous) tumor, located in an area of the brain that releases chemical substances, called the pituitary gland. Over time, having too much growth hormone in the body can alter both appearance and health. Swollen, enlarged hands and feet are some of the most common symptoms. People with acromegaly can also experience sweating, headache, sleep apnea, diabetes, hypertension, and arthritis. It is usually diagnosed in adults aged 30 to 50, but acromegaly can affect people of any age.23
Fibrodysplasia ossificans progressiva (FOP)
Fibrodysplasia ossificans progressiva, or FOP, is an ultra-rare bone condition. It is a genetic disorder caused by a gene mutation (a permanent change to the DNA sequence that makes up a gene)24,25 where the process of normal bone formation cannot be controlled properly leading to extra bone forming where it should not.26 This extra bone formation is permanent, which means the resulting disability can increase over time, affecting a person’s quality of life.27,28
FOP affects as many as 1 to 2 in a million people in the world, but currently about 900 people are known to live with this condition.29,30 Just over half of all people living with FOP are misdiagnosed before they get an accurate diagnosis. Common misdiagnoses include cancer, other tumors, bunions or other rare diseases which can result in unnecessary interventions that might result in extra bone formation.31 People with FOP could benefit from faster diagnosis, allowing them to have the most adapted care.32
Puberty is when a child’s body changes into that of an adult and includes rapid growth of bones and muscles, changes in body shape and size, and development of the body’s ability to reproduce. When puberty begins before the age of eight years in girls and before the age of nine years in boys, it is known as precocious puberty.33
The cause of precocious puberty often can’t be found. Rarely, certain conditions, such as infections, hormone disorders, tumors, brain abnormalities, or injuries, may cause precocious puberty. Treatment for precocious puberty typically includes medication to delay further development with the objective to preserve the adult height.33
Growth disorders are conditions that can stop children from developing normally. There are a number of different types of growth disorders that can affect height, weight and sexual maturity.34
The pituitary gland is a group of cells in the brain that release chemical substances in the body. The pituitary gland makes a hormone which causes bones and other tissues to grow, called growth hormone. Children who don’t make enough growth hormone may not grow as tall as they should.34
There is also a type of growth disorder which is caused by a change to a person’s DNA that causes the body to lack a hormone known as insulin-like growth factor 1.35
Management of these conditions focuses on supporting nutrition intake and development as well as specific growth hormone or IGF-1 therapy, accordingly, in some cases.35
Dedicated websites built with the patient community
Rare diseases are often misunderstood, or completely unknown, and there is little information available for people with rare diseases and their families. Ipsen has worked together with patient organizations and healthcare professionals to co-create dedicated resources for people living with certain rare diseases.
The acromunity website was co-developed with patients and healthcare professionals to provide resources and support for those living with acromegaly. The website also provides information about patients support groups available in individual countries.
Focus on FOP is a website for people living with FOP or their families and caregivers. It features patient stories, videos, and images, as well as more information about the condition. There are also links to national patient organization websites for more information and support.
|Finding information and support
There are a number of websites where you can find reliable information. Patient organization websites are often a good place to start for information and guidance on how to access physical and emotional support.
If you don’t know what local organizations exist, Global and European organizations can often help people to find local resources and groups.
Experiences of living with rare diseases
Although discovering that you are living with a certain condition might feel quite daunting at times, you are certainly not alone. Behind every person, there is a story and we believe you tell that story best.
If you have been affected by any of the experiences or topics discussed in this video, talk to your healthcare professional who may be able to direct you to the additional support you need.