According to a story from BioPortfolio, the biopharmaceutical company Complexa, Inc. recently announced that it has dosed the first patient in its Phase 2 clinical trial which is testing the…
According to a story from PR Newswire, the specialty pharmaceutical company Mallinckrodt plc recently announced the release of interim data from the company's Phase 3 clinical trial. This trial is…
According to a story from Oncology News, a team of researchers affiliated with UNSW Sydney have made a discovery that could have major implications for the rare cancer neuroblastoma, a…
According to a story from Juvenile Arthritis News, a recent study shows that a greater percentage of children with juvenile idiopathic arthritis are participating in school sports activities in the…
The typical treatment for Fabry disease is Fabrazyme which is an enzyme replacement therapy (ERT). This therapy must be administered to the patient every two weeks. A new ERT created by…
What is the Patient Advocate Foundation? The Patient Advocate Foundation (PAF) is a non-profit, formed in 1994 that has one primary aim- eliminate the barriers to treatment too many patients…
Passsage Bio Passage Bio has just announced that they've received 115.5 million dollars to help advance their development of gene therapies. They will begin by using the funds to further…
Cystic Fibrosis Cystic fibrosis (CF) is a rare condition that causes mucus to build up in the lungs and the digestive system. It results in chronic lung infections, lung inflammation, and…
Langerhans Cell Histiocystosis Langerhans Cell Histiocytosis (LCH) Is a rare form of cancer caused by an excessive amount of Langerhan cells in the body. These cells form granulomas which can have a…
Fetal Surgery for Spina Bifida Spina bifida is a rare condition which results in improper spine formation during embryonic development. A gap occurs in the vertebrae causing nerve damage in varying…
According to a publication from Genetic Obesity News, a new genetic mutation has been discovered in an Iranian family that has differing affects on early-onset obesity based on sex. Leptin,…
Hydrocephalus Hydrocephalus is a rare disease which causes a build up of fluid in the ventricles of the brain. The continuous flow of this fluid is how the body prevents the…
Happy Wednesday! Today, we're highlighting an article about gene therapy for MPS, followed by a story about a singer with Achalasia. After that, we have an article about app for…
According to a story from Central Charts, the biopharmaceutical company Inventiva recently announced the release of results from a Phase IIB clinical trial which tested the company's lead product candidate,…
According to a story from BioSpace, the biopharmaceutical company Modis Therapeutics recently announced that the US Food and Drug Administration (FDA) has granted Breakthough Therapy designation for the company's experimental…
A grandmother of four in Maryland has recently become the first cancer patient to complete the experimental CAR-T cell procedure at Georgetown University Hospital. Daisy Diggs 67, who was…
What is Precision Medicine? In essence, precision medicine refers to the idea that in order to establish the best treatment plan for a patient, we must look at them as…
According to a story from prnewswire.com, the biopharmaceutical company Neurotech Pharmaceuticals recently announced that the company had earned Fast Track designation from the US Food and Drug Administration (FDA). This…
The first step in treating rare diseases is accurately diagnosing them. Unfortunately far too many people go years without knowing what is going wrong in their body. Doctors are trained…
An article recently published by the Guardian shared the stories of three families affected by recent breakthroughs in gene therapy treatments. What is Gene Therapy? Gene therapy is an increasingly…
I have lived with pain almost my entire life. Prior to the age of 30, when I developed chronic pain from a neurological movement disorder called dystonia, I experienced pain primarily…
According to a story from Market Screener the biotechnology company Bioblast Pharma has announced a new partnership with Team Sanfilippo a nonprofit foundation which is committed to medical research related…
The Food and Drug Administration (FDA) recently announced its approval of Venetoclax (Venclexta) in combination with either decitabine or azacitidine (hypomethylating agents) or cytarabine (an antimetabolite) to treat adults 75 years…
“Doctor” Onelio Hipolit-Gonzalez has been charged with unlicensed practice of a health care profession and unlawful use of a two-way communication device according to an article published in the Spectrum News of…
Ocugen Pharmaceuticals has just received their first ever orphan drug designation by the FDA for a gene therapy treatment. It's called OCU400 and it's being investigated as a treatment for…
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