How is Niemann-Pick disease treated?

No cure exists for Niemann-Pick disease. No effective treatment is available to people with type A or B. For people with mild to moderate type C, a drug called miglustat (Zavesca) may be an option.

What is NPC condition?

Summary. Niemann-Pick disease type C (NPC) is a rare progressive genetic disorder characterized by an inability of the body to transport cholesterol and other fatty substances (lipids) inside of cells. This leads to the abnormal accumulation of these substances within various tissues of the body, including brain tissue …

Why is VTS 270 being discontinued?

In a letter to families a few weeks ago, the drug manufacturer Mallinckrodt Pharmaceuticals announced they would be discontinuing Adrabetadex because “there is no clear evidence of potential benefit.”

Are there any effective treatments for lipoid pneumonia?

Although there are reports of lipoid pneumonia being successfully treated with corticosteroids, immunoglobulins, and whole-lung lavage, treatment of lipoid pneumonia is not well studied, and the published literature consists only of case reports.

When to take methylprednisone for acute lipoid pneumonia?

Based on clinical history, radiography, and laboratory and bronchoscopic diagnostics, a diagnosis of acute exogenous lipoid pneumonia was made for all five patients. All five patients improved clinically within 24–72 hours after initiation of intravenous methylprednisone (120 mg–500 mg daily).

How is the diagnosis of lipoid pneumonia made?

Diagnosis of this disease requires a high index of suspicion and can be confirmed by demonstration of lipid-laden macrophages in respiratory samples such as sputum, bronchoalveolar lavage fluid or fine-needle aspiration cytology/biopsy from lung lesions. Treatment protocols for this illness are poorly defined. Publication types

How is bronchoscopic segmental lavage used to treat lipoid pneumonia?

In conclusion, a case of exogenous lipoid pneumonia was successfully treated with bronchoscopic segmental lavage therapy.