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Paroxysmal Nocturnal Hemoglobinuria (PNH) is an acquired genetic disorder caused by a mutation in X chromosome gene PIGA (Phosphatidylinositol glycan class A), leading to the destruction of red blood cells, and subsequent release of hemoglobin into plasma. There are mainly three types of PNH cells, namely Normal (type I) stem cells, PNH (type II) stem cell, and PNH (type III) stem cells. Approximately one-half of the patients with PNH die from the disease within 10 years of diagnosis. It occurs equally in men and women, with the peak occurrence in the 30s and middle 50s.

Aplastic Anemia (AA) is a rare and non-cancerous disease that occurs when a person's bone marrow no longer creates enough blood cells. It is believed that aplastic anemia is an autoimmune disorder because the new cells seem to be attacked by the current cells. This limits the amount of new cells that can be created. It is a potential life-threatening hematopoietic stem cell (HSC) disorder with an estimated incidence of 2–3 per million per year. Studies have shown that contact with certain toxins such as benzene and radiation, to viruses such as hepatitis, and to some medications can cause this disease. Aplastic Anemia affects men at somewhat higher rates that it affects women.

The Herpes Simplex Virus, also known as HSV, is an infection that causes Herpes which leads to small, painful, fluid-filled blisters on the skin, mouth, lips, eyes or genitals. This infection is categorized into two types: Herpes Simplex Virus Type-1 (HSV-1) and Herpes Simplex Virus Type-2 (HSV-2). HSV-1 is a highly contagious and common infection that is mainly transmitted by oral contact in or around the mouth, causing cold sores/Herpes Labialis as well as Genital Herpes in some cases. On the other hand, HSV-2 is transmitted sexually (genital-to-genital, oral-to-genital, or genital-to-oral) and perinatally (mother to child). Both HSV-1 and HSV-2 infections are lifelong.

Transthyretin Amyloidosis (ATTR) is an autosomal dominant hereditary disease that occurs due to deposition of variant TTR (transthyretin) protein in humans. It is mainly characterized into two main types i.e. Hereditary ATTR (hATTR) and Wild-Type (wt) ATTR. While Hereditary ATTR is caused by point mutations within TTR allele, wild-type ATTR is caused by the misfolding of Wild-Type TTR. Major prevalence of Hereditary ATTR is observed in the 7MM, and this type has been further sub-classified into Familial Amyloidosis Polyneuropathy (FAP) and Familial Amyloidosis Cardiomyopathy (FAC).

Spasticity is a motor disorder characterized by a velocity-dependent increase in tonic stretch reflexes (muscle tone) with exaggerated tendon jerks with feelings of stiffness and a wide range of involuntary muscle spasms (sustained muscle contractions or sudden movements). It is one of the more common symptoms of Multiple Sclerosis. Spasticity may be as mild as the feeling of tightness of muscles or may be so severe as to produce painful, uncontrollable spasms of extremities, usually of the legs. Spasticity may also produce feelings of pain or tightness in and around joints and can cause low back pain.

Respiratory Syncytial Virus (RSV) is the main viral cause of respiratory tract infection in newborn infants as well as some elderly and high-risk adults with chronic pulmonary disease and are severely immune compromised. Generally, infection is restricted to the upper respiratory tract and not associated with long-term pathology, but progression to a more severe lower respiratory tract infection is frequent. Currently, RSV is considered as the main reason for causing acute lower respiratory infection (ALRI) and a major cause of hospital admissions and death in young children.

The Report "Alpha- Antitrypsin Deficiency - Market Insights, Epidemiology and Market Forecast-2025" provides a comprehensive review of the market trends, drivers, barriers, key company profiles including its business and product developments.

Hereditary Angioedema (HAE) is an autosomal dominant disease which is caused by a deficiency in functional C1 inhibitor. It is a rare hereditary disease which is characterized by painful, unpredictable, recurrent attacks of inflammation and swelling affecting the hands, feet, face, abdomen, urogenital tract, and the larynx. This genetic disease is caused by the production of either inadequate or nonfunctioning C1-Inhibitor protein. The C1-Inhibitor helps in the regulation of the complex biochemical interactions of blood-based systems which are involved in disease-fighting, inflammatory responses, and coagulation.

Multiple sclerosis (MS) is a potentially disabling disease of the brain and spinal cord in which the immune system attacks the myelin sheath. The disease can cause the nerves to weaken or become permanently damaged. Although the exact cause is unknown, the disease is considered to be an autoimmune disease. There are mainly four types of MS namely Relapsing-remitting multiple sclerosis (RRMS), Secondary-progressive multiple sclerosis (SPMS), Primary-progressive multiple sclerosis (PPMS), Benign multiple sclerosis. MS typically begins between the ages of 20 and 40 years and it is the leading cause of non-traumatic disability in young adults.

Food allergy is an allergic disease caused by the hypersensitivity of the immune system food. Food allergy can be caused by various allergic reactions but 90% of allergic responses to food are caused by cow's milk, soy, eggs, wheat, peanuts, tree nuts, fish, and shellfish. Food Allergy can cause itching, swelling of the tongue, vomiting, diarrhea, hives, trouble breathing, or low blood pressure in a person and usually occurs within minutes to several hours of exposure.