Marinus Pharmaceuticals is a pharmaceutical company that works to create treatments for epilepsy and other neuropsychiatric disorders. They are in the process of conducting clinical trials to evaluate the safety…
Karyopharm, a pharmaceutical company based in Newton, Massachusetts, has submitted a New Drug Application to the FDA for their drug XPOVIO, which is meant for the treatment of diffuse large…
Prevail Therapeutics recently submitted an Investigational New Drug application for their gene therapy called PR001. The FDA has recently notified them that this application is active, meaning that Prevail Therapeutics…
Inventiva has recently completed Phase IIa of their clinical trial of odiparcil, a drug made for the treatment of mucopolysaccharidosis type VI (MPS VI). Iventiva is very excited about the…
Copyscape score: 4% Cancer is a disease that affects different people in different ways, and for some people, no existing treatments can effectively treat their cancer. GlaxoSmithKline has recently released…
Copyscape score: 2% Alnylam has recently completed the third phase of their clinical trial of lumasiran, which was created for the treatment of primary hyperoxaluria type 1 (PH1). While they…
Copyscape score: 9% Deondra Clark was unable to have many of the experiences that other children had. Homecoming, prom, sports, and other childhood activities were not a possibility for her,…
For a long time, Catherine Allen went without a diagnose to explain the myriad of symptoms that she experienced. Doctors dismissed her experiences as common feelings in teenagers, when in…
Mustang Bio, a biopharmaceutical company, has been working with St. Jude Children's Research Hospital to develop MB-107, a lentiviral gene therapy to treat X-linked severe combined immunodeficiency. Mustang Bio…
A drug created by Agios Pharmaceuticals, TIBSOVO, was recently granted the Breakthrough Therapy designation by the FDA for the treatment of relapsed or refractory myelodysplastic syndrome (MDS) with an…
The 27th United European Gastroenterology Week took place in Barcelona, Spain in October, at which some of the leading experts in gastroenterology spoke about their field. One of the…
Oncternal Therapeutics is working on TK216, a drug that is the first in its class to treat Ewing sarcoma. They have recently opened enrollment for Phase 1b of their…
Dicerna Pharmaceuticals works in ribonucleic interference (RNAi) therapies as treatments of rare diseases. They have recently been granted the orphan drug designation by the European Commission for their drug, DCR-A1AT.…
New developments in drugs and technology have greatly improved treatment for a variety of diseases, one of them being inflammatory bowel disease. These developments have bettered the lives of…
Mantle cell lymphoma (MCL) is a very rare form of cancer, and oncologists may only see a few cases of it through the course of their careers. Because of…
There is no known cure for sickle cell disease. Treatment is symptomatic and meant to prolong life. While a blood and bone marrow transplant is an option, not everyone…
Parents are often worried about the health of their children; they do not want anything bad happening to their kids. They constantly look out for signs and symptoms of…
Gareth Crabb is a thoughtful and caring six-year-old boy from Swansea, U.K. He is spending his second Christmas in the hospital this year, as he has anaplastic large cell…
Grajevis Bakatunkanda lived in the Democratic Republic of the Congo, where he received treatment for malaria after experiencing weekly pain crisis. The treatment he received did not work, as…
Treatment options for Gorlin syndrome are often surgical, meaning that patients face possible scarring, loss of facial function and structure, and lowered quality of life. PellePharm aims to change…
A new study has found that young people who have inflammatory bowel disease (IBD) are five times more likely to develop an infection than the general population. They also…
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Dystrophic epidermolysis bullosa (DEB) is a condition that has many sub-types, one of which is pretibial. The COL7A1 gene, which is responsible for producing VII collagen, is the cause of…
Amyloidosis is characterized by stiffness and an inability to function in certain organs. Because of the danger this presents, it is important that doctors are able to monitor the…
Autoimmune encephalitis is a group of diseases that was only identified about a decade ago. The newness of this condition makes it difficult to completely understand it. Dr Robb…
Many artists draw inspiration from events in their lives. Dylan Mortimer is no exception, as his art is inspired by his cystic fibrosis. He creates art that mimics lungs,…
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