Rare Disease

Rare Disease

Innovative therapies clinical plan requires understanding Why Immune Cells Extrude Webs of DNA and Protein

February 25th, 2020 | Rare Disease

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 […]

2019 Successful Clinical Plan and Drug Development Stories

January 27th, 2020 | Rare Disease

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, […]

Challenges in autoimmune disease clinical plans

January 3rd, 2020 | Rare Disease

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 […]

PKAN drug fails Phase III trial: How to avoid a similar disaster.

November 26th, 2019 | Rare Disease

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 […]

Inflammation is a recognized cause of Neurodegeneration.

November 21st, 2019 | Rare Disease

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. Boston Biotech Clinical Research has experience in biologics for rare diseases and can help develop a targeted strategy for your needs.

October 31st, 2019 | Rare Disease

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, […]

Do Not Make Mistakes!

September 30th, 2019 | Rare Disease

Could Seamless Adaptive Designs Advance Rare Disease Clinical Trial Design?

July 24th, 2019 | Rare Disease

Gene Therapy for Rare Disorders Europe 2019 – October 15th – 17th, London

July 15th, 2019 | Rare Disease

Drug development success hinges on simplifying Clinical Research

May 16th, 2019 | Rare Disease