Phenylketonuria (PKU) Market Research Report 2030 | Phenylketonuria (PKU) Market

 What is Phenylketonuria?

Phenylketonuria is a rare genetic condition that causes an amino alkanoic acid called phenylalanine to create within the body. Phenylalanine is found altogether in proteins and a few artificial sweeteners.

Phenylalanine hydroxylase is an enzyme your body uses to convert phenylalanine into tyrosine, which your body must create neurotransmitters like epinephrine, norepinephrine, and dopamine. Phenylketonuria is caused by a defect within the gene that helps create phenylalanine hydroxylase.

Phenylketonuria Sign & Symptoms

Newborns with Phenylketonuria initially don't have any symptoms. However, without treatment, babies usually develop signs of Phenylketonuria within a few months.

Phenylketonuria signs and symptoms can be mild and may include:

●     A musty odor in the breath, skin, or urine

●     Neurological problems

●     Eczema

●     Microcephaly

●     Hyperactivity

●     Intellectual disability

●     Delayed development

●     Psychiatric disorders

Phenylketonuria Treatment Options

There is currently no cure for Phenylketonuria, but the condition is controllable through proper diet. The main way to treat Phenylketonuria is to eat a special diet that limits foods containing phenylalanine.

When your infant is old enough to eat solid foods, you need to avoid letting them eat foods high in protein. These foods include eggs, cheese, nuts, milk, beans, and chicken, etc.

Phenylketonuria Epidemiology Insights

The National Phenylketonuria Alliance estimates that there are currently 16,500 people living with Phenylketonuria in the United States.

Germany has the highest number of cases, i.e., 8,317, followed by France with 7,523 cases and therefore the United Kingdom with 6,564 cases. While Spain has the smallest number of cases with 2,490 in 2020.

In 2020, the diagnosed Phenylketonuria prevalent cases in Japan were 1,015.

Phenylketonuria Market Insights

Currently, there is no cure for Phenylketonuria (PKU); however, the prevailing treatment is predominantly through dietary restriction of phenylalanine (Phe) to the minimum required for normal growth, supplemented with specifically designed medical foods. For many years, the only treatment for PKU has been the adherence to the strict low phenylalanine diet for life.

Therefore, the first line of therapy for Phenylketonuria treatment is dietary therapy, where a low Phe diet is supplemented with amino acid formulas, which commences soon after diagnosis. The goal of treatment for PKU is to keep plasma phenylalanine levels within 120–360 µmol/L (2–6 mg/dL), which is achieved through carefully planned and monitored diet. Limiting the child’s intake of phenylalanine, cautiously as it is an essential amino acid. A carefully maintained diet can prevent intellectual disability as well as neurological, behavioral, and dermatological problems. 

Phenylketonuria Emerging Therapy Assessment

Companies across the globe are diligently working toward the development of novel treatment therapies with a considerable amount of success over the years. Key players, such as Synlogic, Censa Pharmaceuticals, Codexis, Homology Medicines, etc., are developing therapies for the treatment of patients with Phenylketonuria (PKU). 

 

Comments

Post a Comment

Popular posts from this blog

Tenosynovial Giant Cell Tumors (TSGCTs) Market Research Report 2030 | Tenosynovial Giant Cell Tumors (TSGCTs) Market

Image
Tenosynovial Giant Cell Tumors   are a type of neoplasm that affects synovial-lined tendon sheaths, synovial joints, and surrounding soft tissue. Locally aggressive benign tissue lesions are defined by synovial lining inflammation in joints and tendons. Overexpression of the colony-stimulating factor 1 receptor is thought to cause these tumors to form. Giant cell tumors of the tendon sheath (GCTTS), pigmented villonodular tumors (PVNTS), extra-articular pigmented villonodular tumors of the tendon sheath, and localized or focal nodular synovitis are all terms used to describe TSGCTs. Tenosynovial Giant Cell Tumors Causes Translocation is an alteration to a chromosome that causes Tenosynovial Giant Cell Tumors. A chromosome segment breaks off and moves around. The cause of these translocations is unknown. The genetic code for making proteins is stored on chromosomes. A protein termed colony-stimulating factor 1 is produced in excess due to the translocation (CSF1). This protein attracts
Read more

Functional Dyspepsia Market Research Report 2030 | Functional Dyspepsia Market

Image
  Functional dyspepsia   is a term for recurring signs and symptoms of indigestion that have no apparent cause. Functional dyspepsia is also called non-ulcer stomach pain or non-ulcer dyspepsia. It's not clear what causes functional dyspepsia. Doctors consider it a functional disorder, which means that routine testing may not show any abnormalities. Hence, it is diagnosed based on symptoms. Functional Dyspepsia Signs and Symptoms  Functional dyspepsia is common and can be long-lasting — although signs and symptoms are mostly intermittent. These signs and symptoms resemble those of an ulcer, discomfort in your upper abdomen, often accompanied by bloating, belching, and nausea, An early feeling of fullness (satiety) when eating, Pain in the stomach that may sometimes occur unrelated to meals, or maybe relieved with meals. Functional Dyspepsia Epidemiology According to an article by Madisch et al. (2018), Functional Dyspepsia is one of the common functional disorders, with a prevalenc
Read more

Fibrodysplasia Ossificans Progressiva Market Research Report 2030 | Fibrodysplasia Ossificans Progressiva Market

Image
  Fibrodysplasia ossificans progressiva   is a disorder in which the skeleton, muscles, and connective tissues, such as tendons and ligaments, are replaced by bone (ossified). This position was to lead to the formation of the bones of the skeleton (extra-skeletal or heterotopic bone) that constrains movement. Fibrodysplasia ossificans progressiva Causes Fibrodysplasia ossificans progressiva is called a mutation in the ACVR1 gene. This gene is involved in bone growth and development. Mutations, and the possibility of a disorderly development. The gene can be inherited from a parent, but in most cases, the, is a new mutation, a person with a less-than-a family history of the disease. As a parent, they have a 50% chance of passing it on to their children. Fibrodysplasia ossificans progressiva Sign and Symptoms The  Fibrodysplasia ossificans progressiva  symptoms begin in the first decade of life with episodes of painful inflammatory soft tissue swellings. Gradually, there occurs restricti
Read more