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There has been a renaissance in gene therapy attempts, spurred partly by the discovery and understanding of novel gene delivery vectors. Adeno-associated virus (AAV) is a non-enveloped virus that may be designed to carry DNA to target cells and has sparked considerable interest in the area, particularly in clinical...

Congenital adrenal hyperplasia (CAH) is a rare inherited autosomal recessive disorder defined by a lack of one of the enzymes required to produce specific hormones. In the United States and Europe, the most prevalent type of CAH, 21 hydroxylase deficiency, affects around 1:10,000 to 1:15,000 people. The estimated C...

Molluscum contagiosum, often known as water warts, is a common superficial skin disease caused by the poxvirus. The molluscum contagiosum virus (MCV) is one of the world's 50 most common diseases. It mostly affects school-aged children, sexually active young adults, and immunocompromised people.  As per our...

Dystrophic epidermolysis bullosa (DEB) is a common skin disease with a chronic history and low healing rates. Despite current management therapies, the disease has the potential to become severe and devastating for many patients over time. Dystrophic epidermolysis bullosa is classified into six types: dominant dyst...

Progressive multifocal leukoencephalopathy is a severe, progressive, and lethal demyelinating disease that presents in immunocompromised states. It is a rare indication, with just over 1.5K PML diagnosed incident cases in the United States, per DelveInsight analysis. It occurs in the setting of predisposing immunos...

Though a rare disease, generalized myasthenia gravis (gMG) still affects around 60,000 people in the US and another 100,000 in EU-5 and Japan. As per our analysis, the total diagnosed prevalent cases of gMG were approx 116K in the 7MM in 2021. These numbers are further estimated to increase by 2032. Moreover, t...