Emerging treatments

Emerging treatments

The recent European Medicines Agency (EMA) approval of the novel ex vivo gamma-retroviral gene therapy is a key milestone for progress in adenosine deaminase-deficient severe combined immune deficiency (ADA-SCID) treatment. The field of genome editing is rapidly progressing providing exciting new options with higher efficacy and improved safety.

... read more

Currently approved and standard of care therapies for ADA-SCID

Currently approved and standard of care therapies for ADA-SCID

There are currently three main management options to treat ADA deficiency; haematopoietic stem cell transplantation (HSCT), enzyme replacement therapy (ERT) and gene therapy (GT).

... read more

Newborn screening

Newborn screening

The aim of newborn screening (NBS) is to identify infants who present as healthy at birth but have an underlying condition associated with severe morbidity and mortality. In many countries, population-wide NBS is used as a successful technique for diagnosing a wide range of clinical conditions.

... read more

Biology of ADA-SCID

Biology of ADA-SCID

ADA deficiency is an inherited disorder that damages the immune system and causes SCID. People with SCID lack virtually all immune protection from bacteria, viruses and fungi. They are prone to repeated and persistent infections that can be very serious or life-threatening. These infections are often caused by opportunistic organisms that ordinarily do not cause illness in people with a normal immune system.

... read more