Is SMA rare?
Is SMA rare?
Most people have never heard of spinal muscular atrophy (SMA), a rare genetic disease that affects approximately 1 in 6,000 babies born worldwide each year. Innovative research may produce treatments for patients suffering from SMA and other rare diseases.
Is cervical dystonia rare?
A rare disorder that can occur at any age, cervical dystonia most often occurs in middle-aged people, women more than men.
How common is spinal muscular atrophy?
Approximately one in 6,000 babies born have SMA, and about one in 40 people carry a copy of the altered gene that causes the condition (although they do not have the condition themselves). This is known as being a genetic carrier of the condition.
Why SMA injection is so costly?
At $2.125 million, it is touted to be the world’s most expensive drug. The price, according to industry sources, is due to the low incidence of the disease, which means a smaller number of patients, as well as the significant costs in the research in gene therapy and the drug development process.
Is cervical dystonia inherited?
Primary cervical dystonia is associated with a hereditary component in approximately 12% of cases, and it may possibly be linked to previous neck injury.
Is cervical dystonia life-threatening?
Is dystonia life-threatening? For the overwhelming majority, dystonia does not shorten life expectancy and is not fatal. In severe generalized dystonia that affects many body areas, problems can arise that are secondary to the dystonia and require emergency care.
What percentage of the population has SMA?
Roughly 1 in 50 (or approximately 6 million) people in the United States carries a copy of the mutated SMN1 gene responsible for SMA. These people are called carriers.
How many SMA cases are there in India?
SMA affects approximately one in 10,000 live births globally and one in 7,744 live births in India. Without treatment, babies with the most severe form of SMA, Type 1, cannot sit unsupported.