It was discovered by a team led by Koen Andries at Janssen Pharmaceutica.Bedaquiline was approved for medical use in the United States in 2012.It is manufactured by Johnson & Johnson (J&J), who sought accelerated approval of the drug, a type of temporary approval for diseases lacking other viable treatment options. By gaining approval for a drug that treats a neglected disease, J&J is now able to request expedited FDA review of a future drug.When it was approved by the FDA on 28 December 2012, it was the first new medicine for TB in more than forty years.In 2016, the WHO came under criticism for recommending it as an essential medicine.The WHO TB program director has pointed out that Janssen will donate $30 million worth (30,000 treatment courses) of bedaquiline over a 4-year period. References
External links
"Bedaquiline". Drug Information Portal. U.S. National Library of Medicine.
Aase syndrome or Aase–Smith syndrome is a rare inherited disorder characterized by anemia with some joint and skeletal deformities. Aase syndrome is thought to be an autosomal dominant inherited disorder. The genetic basis of the disease is not known. The anemia is caused by underdevelopment of the bone marrow, which is where blood cells are formed. It is named after the American paediatricians Jon Morton Aase and David Weyhe Smith, who characterized it in 1968. Signs and symptoms
Among the presentation are:
Mildly slowed growth
Pale skin
Delayed closure of fontanelles (soft spots)
Narrow shoulders
Triple jointed thumbs, absent or small knuckles, decreased skin creases at finger joints
Inability to fully extend the joints from birth (congenital contractures)
Cleft palate
Deformed ears
Droopy eyelids

Complications
Complications related to anemia include weakness, fatigue, and decreased oxygenation of the blood. Decreased white blood cells alter the bodys ability to fight infection. If a heart defect exists, it may cause multiple complications (depending on the specific defect). Severe cases have been associated with still birth or early death. Cause
Some cases of Aase syndrome (45%) have been shown to be inherited, and are due to a change in one gene which makes ribosomal proteins. However, many cases are not inherited and occur without a known cause. Diagnosis
A CBC (complete blood count) will show anemia and a decrease in the white blood cell count. An echocardiogram may reveal heart defects (ventricular septal defect is most common). X-rays will show skeletal abnormalities as described above. A bone marrow biopsy may be performed.