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The drug was granted priority review designation by the FDA before being approved in the US on 6 September 2019, to slow the rate of decline in pulmonary function in patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD). [38] [39] It is the first FDA-approved treatment for this rare lung condition. [38]
ACD is a rare disease. As of 2011, about 100 cases had been reported. It is likely an under-recognized cause of death shortly after birth because diagnosis requires microscopic examination of lung tissue or specialized genetic testing, or death can be attributed to severe heart or intestinal congenital abnormalities which frequently occur in ACD.
Serious side effects including hypersensitivity and swelling have been observed in renal transplant patients. [22] While sirolimus was considered for treatment of LAM, it received orphan drug designation status because LAM is a rare condition. [22] The safety of LAM treatment by sirolimus in people younger than 18 years old has not been tested.
Merck said its drug met the main goal of significantly reducing the time to disease worsening, lung transplantation or death in a late-stage study of 172 patients with advanced stages of the ...
Childhood interstitial lung disease, sometimes abbreviated as ChILD, is a family of rare chronic and complex disorders that affect the lungs of children. [1] In the lungs, these disorders affect the interstitium, which is the space around the alveoli. The alveoli are the air sacs of the lungs. [2] For these disorders, the alveoli are typically ...
It is a type of interstitial lung disease. [3] People often benefit from pulmonary rehabilitation and supplemental oxygen. [1] Certain medications like pirfenidone (Esbriet) or nintedanib (Ofev) may slow the progression of the disease. [2] Lung transplantation may also be an option. [1] About 5 million people are affected globally. [8]
Pulmonary Alveolar Microlithiasis patients, families, and caregivers are encouraged to join the NIH Rare Lung Diseases Consortium Contact Registry. This is a privacy protected site that provides up-to-date information for individuals interested in the latest scientific news, trials, and treatments related to rare lung diseases. [citation needed]
Pulmonary drug delivery is mainly utilized for topical applications in the lungs, such as the use of inhaled beta-agonists, corticosteroids and anticholinergic agents for the treatment of asthma and COPD, the use of inhaled mucolytics and antibiotics for the treatment of cystic fibrosis (CT) and respiratory viral infections, [1] and the use of inhaled prostacyclin analogs for the treatment of ...
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