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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.

Inflammatory bowel disease (IBD) refers to a group of chronic inflammatory diseases, including ulcerative colitis and Crohn’s disease, that cause swelling of the tissues in the digestive tract.¹⁶ It occurs when the body’s natural defense system mistakenly attacks healthy bowel cells.¹⁷ Symptoms typically flare up and then recede and often include diarrhea, stomach pain, rectal bleeding, fatigue, and weight loss. While IBD can be mild for some, for others, it can cause disability and lead to life-threatening complications.¹⁶

Rheumatoid arthritis is a chronic, inflammatory condition in which the body’s immune system attacks its own tissues, causing significant inflammation in joints and other organs. It most commonly affects small joints in the wrists, hands, and feet, causing joint pain, stiffness, swelling, and decreased flexibility, but can also affect larger joints and organs such as the skin, eyes, heart, lungs, and blood vessels. Despite medical advancements, rheumatoid arthritis can still cause long-term damage and increase the risk of heart disease.^18,19