Myositis and Cystic Fibrosis are rare genetic diseases related to the inflammation and damage of different tissues. Myostisis is characterized by muscular inflammation leading to muscle weakness, painful and/or aching muscles, tripping or falling, and extreme tiredness after walking or standing. Myositis might be caused by an underlying problem with the immune system, where it mistakenly attacks healthy tissue. Most patients respond to the steroid and immunosuppressive therapy, and a carefully-controlled exercise. In some more severe cases, patients develop breathing and swallowing problems and it can be related to different types of cancer.
Cystic fibrosis is characterized by chronic inflammation and damage to the lungs. It can also affect the pancreas, liver, kidneys and the gut. The patients have shown recurring chest infections, weight loss, frequent-wet-sounding coughs, diarrhea and shortness of breath. Complications might include weakened bones, diabetes and liver issues.
Unfortunately, there is not a known cure for cystic fibrosis. However, some palliative treatments are available to help control the symptoms, prevent complications, and manage the quality of life.This highlights the need to develop treatments that can help patients with these orphan diseases.
References and patients organizations: