A new Phase 1 clinical trial for celiac disease has just begun dosing patients. This trial is being run by Anokion SA. Celiac Disease Celiac disease is an autoimmune condition…
Pyoderma gangraenosum (PG) is a rare auto inflammatory disease which affects the skin. It unfortunately has no currently approved therapies. However, recent preliminary results from a Phase 2a trial have…
Rare Disease Institute The Rare Disease Institute (RDI) is a new initiative aimed at improving collaboration between rare disease stakeholders. The aim is to facilitate faster diagnosis and better care…
The Institute for Gene Therapies (IGT) is a new development, aimed at improving the speed of development and accessibility of gene therapies. Gene Therapy Many rare diseases are caused by…
Why are rare diseases so difficult to study? Is it just their rarity? Or is it also industry challenges? Unfortunately, industry has a lot to do with it. Clinical Trial…
When your child receives a rare diagnosis that needs instant care, you don't have time to contemplate finances and plan out your budget. You just have to act. That was…
Clinical trials and healthcare as a whole are being transformed by new technology. It's essential for clinical trial sponsors to be aware of the new options available to them, and…
Alyla Annac When Alyla Annac was just 19 years old her father passed away from heart failure. He had suffered 3 heart attacks and doctors said there was nothing else…
Sedation Versus Sleep Sleep and sedation are not synonymous terms, despite the fact that they are often used interchangeably in the hospital setting. The biggest difference? Sleep is always beneficial…
Christine Wright Christine Wright was an active, athletic, healthy mother of three. But in 2007 she started exhibiting strange symptoms that no one could give her an answer to. She…
During the 1990s, researchers had a dream that by 2020, every individual would have access to their own genome. This would aid in their diagnosis and treatment as soon as…
Without understanding the cause of disease it is difficult to understand how to most effectively care for patients. Instead of addressing the underlying cause of disease, physicians are forced to…
Lennox-Gastaut syndrome (LGS) is a form of epilepsy. Most patients with this condition are resistant to current treatments and experience seizures throughout the duration of their life. They may face…
A recent study led by Morten Aagaard Nielsen and Tue Wenzel Kragstrup, who both work at Aarhus University in Denmark, hopes to help physicians determine the best personalized treatment for…
AskBio Asklepios BioPharmaceuticals (AskBio) was first founded in 2001. It is based out of North Carolina. This company is dedicated to the research of gene therapies as potential therapeutic options…
Hypotonia Ataxia, and Delayed Development Syndrome Hypotonia, Ataxia, and Delayed Development Syndrome (HADDS) is caused by mutations in the EBF3 gene which is found on chromosome 10q26. Its discovery just…
Angiosarcoma Angiosarcoma is a rare condition caused by a tumor within the endothelial cells. These cells line the blood vessels in the body, and therefore the tumors can occur practically…
Methylmalonic Acidemia Methylmalonic acidemia (MMA) is a rare disease affecting the metabolic system. 60% of all cases are due to a deficiency in MUT, a mitochondrial enzyme, which causes acids to…
Spinal muscular atrophy (SMA) currently has two approved treatment options. Just 3.5 years ago this number was zero and in another year or so, researchers are hoping to increase it to…
Phase 3 Trial A Phase 3 trial run by Pharnext examining the safety and the efficacy of an investigative therapy called PXT3003 began in December of 2015. It enrolled 323…
Lisa Christopher-Stine is the Director of the Myositis Center at Johns Hopkins. She recently shared her biggest tips for patient care and some updates from the field, hoping to change…
Hereditary Angioedema Hereditary angioedema (HAE) is a rare disease which causes swelling episodes throughout the body, including the airways, gastrointestinal system, and extremities. Most commonly, HAE is caused by a…
Ipsen is a pharmaceutical company based out of Paris. Sadly, they have just announced that they have pressed pause on not one but two studies for fibrodysplasia ossificans progressiva (FOP). FOP…
The State of PTO Of all of the industrialized countries in the world, the United States is the only one that in the year 2020 still doesn't guarantee that workers…
Proteostasis Therapeutics has just announced results from their ex-vivo study of PTI CFTR modulators using organoids from patients living with cystic fibrosis (CF). These organoids are genetically identical, and have…
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