Pediatric Rare diseases and Rare Pediatric Disease Designation FDA will award priority review vouchers to sponsors of certain rare pediatric disease product applications that meet the criteria specified. Section 529 of the FD&C Act is intended to encourage development of new drug and biological products for the prevention and treatment of rare pediatric diseases. Section […]
Scientists launched studies in search of genes that could explain why some people infected with SARS-CoV-2 get really sick, while others have only mild symptoms. We know that chronic health conditions—such as hypertension and diabetes can play a role, but there are also evidences that people’s genes can influence how their bodies react to other […]
The gut microbiome serves many useful functions in the body, but it can also rev up the immune system in harmful ways. Zit has been pustulated that Diet can influence the microbiome and the mucosal immune response. In the paper: “Diet modulates colonic T cell responses by regulating the expression of a Bacteroides thetaiotaomicron antigen,” Sci Immunol, 4:eaau9079, […]
Today, it is widely accepted that NETs have both a protective and a pathological impact on the host. When neutrophils encounter pathogens, not only engage in phagocytosis and degranulation, they also release neutrophil extracellular traps (NETs) In CANCER, NET-associated proteins lead to reawakening of dormant cancer cells and convert them to proliferating metastatic cells. In […]
Gene therapies for disorders such as X-linked Severe Combined Immunodeficiency (SCID, sometimes known as “bubble boy disease”), and Spinal Muscular Atrophy (SMA) have, for the first time, shown remarkable safety and efficacy results in clinical trials. FDA approved Trikafta, a gene-based therapy creating hope for children with cystic fibrosis and Zolgensma, a gene therapy drug, […]
Autoimmunity occurs when the body is unable to differentiate “self” from “non-self” which results in overactive immune response against own cells and tissues. Autoimmune diseases affect 5 %-8% of the population; 78% affected are females. Low level autoimmunity is normal. Over 80 conditions linked to autoimmunity have been identified and 15 diseases directly linked to […]
Retrophin’s experimental PKAN drug fosmetpantotenate fails phase III trial Pantothenate kinase-associated neurodegeneration (PKAN) is a rare disease characterized by a progressive neurodegenerative disorder and buildup of iron in the brain which is estimated to affect up to 5000 patients worldwide. PKAN is inherited in an autosomal recessive manner and is caused by mutations in the […]
Neuroinflammation is inflammation of the nervous tissue. It may be initiated in response to a variety of cues, including infection, traumatic brain injury, toxic metabolites, or autoimmunity. In the CNS, including the brain and spinal cord, microglia are the resident innate immune cells that are activated in response to these cues. The CNS is typically an immunologically privileged site because peripheral […]
Biologic treatments show promise in providing clinical solutions to a variety of diseases Due to biological’s unique characteristics, study designers may find that results in pre-clinical trials do not predict acute, chronic and late onset immunogenicity and safety. In Addition, the biological orphan has a long PK half-life of months such as with any mAb, […]
