Zolgensma

The cell and gene therapy market has seen a revolutionary transition in recent years, with advancements in scientific research and novel methods of treatment driving a rise in development activities. This has resulted in an increase in the number of cell and gene therapy choices available to patients suffering from...

By bringing life-changing benefits to people with rare diseases and cancers, cell and gene therapies are reshaping the field of medicine. These treatments have recently gained tremendous publicity for meeting long-standing unmet needs and being extremely costly. But high production costs are limiting patient access...

In recent years, the development of cell and gene therapies has provided a new avenue for the treatment of rare disorders. This article will discuss the current and future scenarios of cell and gene therapies in rare disorders with cancer indications as an exception, in Japan. Cell therapy is a treatment in which l...

Drug pricing is one of the hottest topics in the healthcare segment. Several arguments have been put forward by people, governments, healthcare companies, and other organizations in favor of and against expensive medicines. Some of the drugs are so expensive that they are nearly out of reach of the common people. H...

CNS disorders are a wide-ranging set of disorders in which the brain loses its normal functioning, limiting everyday ability to function. These may arise from various reasons-some are inherited, some result from damage, and some from infections. It contributes to the highest disability-adjusted life years. The curr...

Bluebird declares the price of its gene therapy- Zynteglo Bluebird Bio has set the price of its gene therapy Zynteglo at USD 1.8 million. Zynteglo, so far, has only been approved for treating rare blood disorders in the Europe Union. The full price of Zynteglo can be dispersed out for five years, at approx...

The U.S. FDA recently approved the much talked about Novartis’s drug Zolgensma for treating a life-threatening condition Spinal Muscular Atrophy (SMA).  SMA is a neuromuscular disorder that worsens the muscles over time. It happens due to a mutation in the SMN1 gene that results in the deficiency of an SMN pro...