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cystic fibrosis


On July 2, 2015 the FDA announced its approval for the potent duo of Lumacaftor and Ivacaftor to treat patients suffering from Cystic Fibrosis (CF). Coined Orkambi, which is marketed by Vertex Pharmaceuticals, this effectual treatment is specifically approved for patients ages 12 and older who have the homozygous F508 mutation of the CF transmembrane[…]

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Known as the brand name Cystic Fibrosis drug Kalydeco, marketed by Vertex Pharmaceuticals, Ivacaftor recently received an MAA line extension. As of June 2015 children ages two through five who have specific mutations in the CFTR gene may be administered Ivacaftor as therapy. As a unique, tolerable and effectual treatment Ivacaftor acts as a novel[…]

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Recently revealed results from a successful Phase 3 study combining Ivacaftor {Kalydeco} and Lumacaftor {VX-809} are extremely positive for patients with a common Cystic Fibrosis {CF} mutation. Patients who have the mutation of Cystic Fibrosis, which entails two copies of the F508del, have demonstrated in Phase 3 clinical trials an effective response to treatment with[…]

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The end of June 2014 brought exciting news for Insmed’s liposomal Amikacin, known as the brand name Arikace, with a Breakthrough Status award from the FDA. Amikacin was specifically designated Breakthrough Status for treatment refractory nontuberculous mycobacterial lung disease in adult patients. As an inhaled antibiotic Amikacin is effectual for treating chronic nontuberculous mycobacterial lung[…]

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Ataluren, formerly known as PTC124 appears to be a viable treatment for Cystic Fibrosis. This oral compound targets CFTR, the protein product and faulty gene believed to be the cause of Cystic Fibrosis. Ataluren also appears to be particularly effective for patients with Cystic Fibrosis (CF) nonsense mutation. This genetic mutation interrupts the CFTR protein[…]

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As an aminoglycoside antibiotic effective for treating a wide variety of infections, Tobramycin CAS# 32986-56-4 is an integral medication that affects a vast number of patients. Tobramycin is particularly effectual for treating Gram-negative infections and certain Pseudomonas. The recent shortage of this crucial antibiotic has been felt for patients who take Tobramycin, either as an[…]

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As a genetic disorder characterized by progressive muscle degeneration and weakness, Duchenne muscular dystrophy, or DMD is a virulent form of muscular dystrophy. The absence of dystrophin, which is a protein that helps to keep muscle cells intact, is the cause of this genetic condition. Duchenne muscular dystrophy is one of the nine types of[…]

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Somatropin is a human growth hormone, also known in the pharmaceutical and medical community as hGH. The brand, Norditropin, which is marketed by Novo Nordisk, has a patent expiration of December 16, 2015. As an injectable treatment for children who have growth failure, either due to low or no growth hormone, somatropin is safe and[…]

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