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Nontuberculous mycobacterial (NTM) lung disease is a rare and serious disorder most commonly caused by a group of bacteria called Mycobacterium avium complex (MAC).¹ People who have bronchiectasis, COPD, and asthma are at greater risk of getting NTM lung disease.^2,3,4

Nontuberculous mycobacterial (NTM) lung disease is a rare and serious disorder most commonly caused by a group of bacteria called Mycobacterium avium complex (MAC).¹ People who have bronchiectasis, COPD, and asthma are at greater risk of getting NTM lung disease.^2,3,4

Non-cystic fibrosis bronchiectasis (NCFBE) is a chronic pulmonary disorder in which the bronchi become permanently dilated due to a vicious cycle of inflammation and complications from prior infections.^5,6 The condition is marked by frequent pulmonary exacerbations requiring antibiotic therapy and/or hospitalizations.

CRS without nasal polyps is a chronic inflammatory process of the mucus membranes inside the sinuses. The most common symptoms include a decreased sense of smell and facial pain.⁷

HS is a chronic relapsing inflammatory disorder, characterized by painful, inflamed, and swollen lesions affecting hair follicles, often in the armpits, groin, and skin folds.⁸

Interstitial lung diseases (ILD) comprise a large group of diseases that cause fibrosis (scarring) of the lungs. Pulmonary hypertension (high blood pressure in the lungs) is a common and important complication of several ILDs that is associated with reduced exercise capacity and poor prognosis.^9,10 PH-ILD is also known as WHO Group 3 Pulmonary Hypertension.

Pulmonary arterial hypertension (PAH) is a serious, progressive, rare disease involving narrowing and constriction of the pulmonary arteries that carry blood from the right side of the heart to the lungs.¹¹ PAH is also known as WHO Group 1 Pulmonary Hypertension.

DMD is caused by a genetic mutation that prevents the body from producing dystrophin, a protein that muscles need to work properly. Without it, muscle cells become damaged and weaken.¹² DMD is the most common childhood onset form of muscular dystrophy and affects males almost exclusively.¹³

ALS is a progressive neurological disease that affects the nerve cells in the brain and spinal cord that control voluntary muscle movement and breathing. As these nerve cells degenerate, the body’s muscles begin to weaken and waste away. Eventually, the brain loses its ability to control voluntary movements like walking, talking, chewing, and breathing. Most people with ALS die of respiratory failure within three to five years from initial symptom onset.¹⁴

Stargardt disease is a rare genetic eye disease caused by the buildup of fatty material on the macula, the small part of the retina needed for sharp, central vision.¹⁵ The disease leads to the loss of central vision in both eyes, typically beginning in childhood. Today, there are no treatments available for the disease, and management options focus on optimizing patients’ remaining sight.

ASA is a rare genetic disorder characterized by a deficiency of the enzyme argininosuccinate lyase (ASL), which plays a role in the breakdown and removal of nitrogen in the body—a process known as the urea cycle. The lack of ASL results in excessive ammonia in the blood, which damages or inhibits the functions of neurons. The disease typically presents in infants and is marked by vomiting, refusal to eat, progressive lethargy, and coma.¹⁶

Chronic refractory gout is a rare form of gout, which can cause chronic arthritis; swelling, redness, and intense pain in the joints; and lumps around the hands, elbows, and other body parts, known as tophi. It is caused by the buildup of uric acid in the body, either due to over-production of uric acid or the inability of the kidneys to remove it.¹⁷

AT is an inherited, fatal childhood disorder primarily driven by mutations in the ataxia-telangiectasia mutated (ATM) gene, which is involved in DNA damage repair. Symptoms vary between individuals but typically involve degeneration across a range of neurological and neuromuscular functions, impaired metabolic and immune system functions, and an increased risk of cancer. People with AT often require significant support and face a significantly reduced life expectancy.¹⁸