Scleroderma (systemic sclerosis [SSc]) is a rare progressive autoimmune disorder characterized by skin fibrosis, systemic inflammation, and varying degrees of vasculopathy that manifest as Raynaud…
Idiopathic pulmonary fibrosis (IPF), although a rare disease, is one of the most common interstitial lung diseases (ILDs). It is characterized by an irreversible loss of lung function, leading to…
Introduction: NASH is characterized by a buildup of excess lipid (steatosis) in the liver, resulting in inflammation and eventually liver damage, leading to fibrosis and subsequently cirrhosis and…
NASH has the potential to be a large and lucrative therapy market owing to the prevalence of the disease and the lack of approved agents. Patients with NASH are at risk of liver fibrosis,…
Nearly 35 years after the Orphan Drug Act of 1983 (ODA) became law in the United States, private investment in the development of drugs for orphan diseases continues unabated. Although the growing…
Progress in R&D and technology, regulatory and monetary incentives for advancing the development of treatments for rare diseases, and ongoing focus by the pharmaceutical market on this space are…
Cystic fibrosis (CF) is a genetic disease caused by any one of the more than 2,000 mutations identified in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. CFTR mutations lead…
Idiopathic pulmonary fibrosis (IPF), although a rare disease, is one of the most common interstitial lung diseases (ILDs); IPF is characterized by irreversible loss of lung function leading to high…
Cystic fibrosis (CF) is a genetic disease affecting chloride transport in a variety of tissues, most notably the lungs and pancreas. It is caused by mutations in the cystic fibrosis transmembrane…
Scleroderma (systemic sclerosis [SSc]) is a rare progressive autoimmune disease characterized by skin fibrosis and varying degrees of vasculopathy manifesting as Raynaud’s disease and, often,…
Idiopathic pulmonary fibrosis (IPF), although a rare disease, is one of the most common interstitial lung diseases (ILDs) and is characterized by irreversible loss of lung function leading to high…
This detailed, expanded analysis explores key access and reimbursement topics in the rare disease space in the United States, such as the approaches managed care organizations take to control both…
Non-alcoholic steatohepatitis (NASH) is a common liver disease that can progress to liver fibrosis, cardiovascular disease, and cancer. In the absence of any approved treatments for it, there is a…
Cystic fibrosis (CF) is a genetic disease affecting chloride transport in a variety of tissues, most notably the lungs and pancreas, and is caused by mutations in the cystic fibrosis transmembrane…
Nonalcoholic fatty liver disease (NAFLD) is caused by a buildup of fat in the liver. A proportion of NAFLD sufferers will also experience injury to their hepatocytes, which can lead to nonalcoholic…