Rare
Diseases
Diseases that affects a small percentage of the population
Advancing Care for Rare Diseases
At Menagen, we are committed to improving the lives of patients with rare diseases by providing innovative treatments that address significant unmet medical needs. Our focus extends to conditions that require specialized therapies and dedicated patient support.
Our Areas of Focus in Rare Diseases
Pulmonary Arterial Hypertension (PAH)
A rare and progressive disease affecting the arteries in the lungs and heart.
Pulmonary Arterial Hypertension
Pulmonary arterial hypertension (PAH) is one form of pulmonary hypertension (high blood pressure in your pulmonary arteries). Symptoms may not appear until the condition has gotten worse.
Pulmonary arterial hypertension strains the right side of your heart, which pumps oxygen-poor blood to your lungs. This strain can lead to right-sided heart failure.
Plus, PAH slows down blood flow between your heart and lungs. This means less blood can enter your lungs to gain fresh oxygen. As a result, blood flow to the rest of your body also slows down. So, your organs and tissues can’t get enough oxygen.
Without treatment, PAH can be fatal.
Hereditary Tyrosinemia Type I
A metabolic disorder that requires lifelong management.
Hereditary Tyrosinemia type I
Tyrosinemia type I is a rare autosomal recessive genetic metabolic disorder characterized by lack of the enzyme fumarylacetoacetate hydrolase (FAH), which is needed for the final break down of the amino acid tyrosine. Failure to properly break down tyrosine leads to abnormal accumulation of tyrosine and its metabolites in the liver, potentially resulting in severe liver disease. Tyrosine may also accumulate in the kidneys and central nervous system.
Cystic Fibrosis
A genetic disease impacting respiratory and digestive health.
Cystic Fibrosis
Cystic fibrosis (CF) is a genetic disease that causes sticky, thick mucus to build up in your organs, blocking and damaging them. Many people think of CF as a lung disease because it affects your lungs and airways, which can make it hard to breathe and cause frequent infections. But it’s called cystic fibrosis because it also causes cysts and scarring (fibrosis) in your pancreas. This damage, plus the thick mucus, can block ducts that release digestive enzymes, making it hard to get nutrients from your digestive tract. CF can also affect your liver, sinuses, intestines and sex organs.
The mucus that lines your organs and body cavities, such as your lungs and nose, is thin and watery. In people with CF, a change in a gene (genetic mutation) leads to low levels of certain proteins, or proteins that don’t work properly. Because of these faulty proteins, minerals that move water into your mucus (which thins it out) get trapped inside cells, leaving the mucus thick and sticky.
People with cystic fibrosis are born with it. It’s a lifelong illness that gets more severe over time. Most people with CF don’t live as long as people without it.
Hereditary Angioedema (HAE)
A rare condition causing unpredictable swelling attacks.
Hereditary Angioedema (HAE)
Hereditary angioedema (HAE) is a rare, genetic condition that causes episodes of swelling in different parts of your child’s body, including their face, hands or feet. Sometimes, swelling can happen in areas you can’t see, like in the tissues that line your child’s airways or digestive organs.
This swelling occurs because fluid leaks out of tiny blood vessels in your child’s body. The fluid builds up in the nearby tissues, which can prevent blood or lymph from moving along as it should. Episodes of swelling and related symptoms (HAE attacks) can be unpredictable and range in severity from mild to life-threatening.
“Angio-” refers to blood vessels, and “-edema” is the medical term for swelling due to fluid buildup within bodily tissues. There are many types of angioedema, each with different causes. HAE is one of the rarer types. It’s congenital, which means a person is born with it.
Cardiac Myopathy
A group of heart diseases affecting the heart muscle’s ability to pump blood effectively.
Cardiomyopathy
Cardiomyopathy is a disease that affects your myocardium (heart muscle). Cardiomyopathy can make your heart stiffen, enlarge or thicken and can cause scar tissue. As a result, your heart can’t pump blood effectively to the rest of your body.
In time, your heart can weaken and cardiomyopathy can lead to heart failure. Treatment can help. Some people with cardiomyopathy eventually need a heart transplant.Pulmonary arterial hypertension strains the right side of your heart, which pumps oxygen-poor blood to your lungs. This strain can lead to right-sided heart failure.
Plus, PAH slows down blood flow between your heart and lungs. This means less blood can enter your lungs to gain fresh oxygen. As a result, blood flow to the rest of your body also slows down. So, your organs and tissues can’t get enough oxygen.
Without treatment, PAH can be fatal.
Our Current Brands:
Nitisinone Menagen®
Nitisinone 2 mg Capsules
Therapy for Hereditary Tyrosinemia Type I.
Ivacaf®
Ivacaftor 150 mg Tablets
Targeted treatment for Cystic Fibrosis
Icatigen®
Icatibant 30 mg / mL PFS
A solution for managing Hereditary Angioedema.
Ambrisentan/tadalafil
Treatment for Pulmonary Arterial Hypertension
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