Assessing How Emerging Therapies Will Transform...

FDA Approves Yescarta as a First CAR T-cell Therapy for Initial Treatment of R/R Large B-cell Lymphoma Until now, existing CAR-T therapies have been reserved for patients with blood cancer who have tried multiple treatments. The FDA has approved Yescarta, a CD19-directed CAR-T therapy developed by Gilead Science...

Sanofi’s Rare Disease Drug Xenpozyme Wins World’s First Approval in Japan Sanofi’s Xenpozyme, also known as olipudase alfa, the world's first therapy for the rare disease Niemann-Pick type A/B and B, has been approved for the treatment of children and adults with non-CNS manifestations of Acid Sphingomyelinase D...

Fabry Disease is an inherited lysosomal storage disease caused by a nonfunctional or partially functional enzyme called alpha galactosidase A (α-Gal A). Decreased activity of α-Gal A in lysosomes results in the accumulation of enzyme substrates (Gb3 and lyso-Gb3) which cause cellular damage in tissues throughout th...

Amyotrophic Lateral Sclerosis (ALS) or Lou Gehrig’s disease is a rare, progressive, and fatal neuromuscular disease characterized by degeneration of upper and lower motor neurons. While it is associated with a mortality rate of 50% within 30 months of Amyotrophic Lateral Sclerosis symptoms onset, there is still no ...

Parkinson's disease (PD) is the second most prevalent neurogenerative disorder, with a growing global prevalence due to population aging. Despite increased awareness and significant research advancement, Parkinson’s disease treatment options include dopamine replacement therapy, symptomatic therapies that have sign...

Adrenal Crisis refers to overwhelming and life-threatening adrenal insufficiency. The most common signs of Adrenal Crisis are shocking (very low blood pressure with a loss of consciousness), dehydration, and an imbalance of sodium and potassium levels in the body. In some cases, shock is then preceded by several ot...