After three years, patients with Leber hereditary optic neuropathy (LHON) who had been treated with LUMEVOQ experienced sustained safety and efficacy. According to a press release from GenSight Biologics,…
In 2019, Alnylam Pharmaceuticals received FDA approval for Givlaari, the first RNAi therapy for patients with acute hepatic porphyria. Although some were initially worried about the therapy's safety and…
Scientists from the Universities of Edinburgh and Cambridge in collaboration with a research center in Germany have analyzed a DNA-damaging chemical that has given scientists a better understanding of the…
If you could take a vaccine to prevent disease progression, would you? Well, researchers recently tested the efficacy of such a vaccine for patients with Parkinson's disease. The experimental and…
This morning, SynAct Pharma AB announced in a press release that they began a Phase II clinical trial to determine the efficacy and safety of AP1189 on treating patients with…
This week, UbiVac and Bristol Myers Squibb announced their clinical trial collaboration in a Biospace press release. The two companies, in a Phase 1b trial, look to study whether combining…
Currently, up to 5% of American citizens have nonalcoholic steatohepatitis (NASH), a liver disease resulting in damage and inflammation. But there's no approved treatments, presenting an unmet need in…
According to BioSpace, biotechnology company Argenx recently announced positive results from their Phase III ADAPT clinical trial. The company, who seeks to find breakthroughs in immunology and translate them…
It all began with an email in 2019. Matt Might, director of the Hugh Kaul Precision Medicine Institute, shot his thoughts over to neurosurgery resident Matt Davis, whose 3-year-old son…
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…
Big development for those in the primary hyperoxaluria type 1 (PH1) community! Alnylam Pharmaceuticals just announced that its Phase 3 study of lumasiran, an investigational therapy for the treatment of primary…
Some promising news for the congenital adrenal hyperplasia (CAH) community! Spruce Biosciences is one step closer to a treatment for CAH after new phase 2a data showed the drug reduced…
Eosinophilic Esophagitis (EoE) Eosinophilic esophagitis (EoE) is a rare and chronic disorder which affects the digestive system. Eosinophils are a type of white blood cell that help fight infections. This…
According to a publication from BusinessWire, the Maryland-based pharmaceutical company OncoImmune, Inc. has announced successful phase 2 trial results for its acute graft versus host disease (GVHD) drug CD24Fc. Graft…
Cold agglutinin disease (CAD) occurs when the body's antibodies destroy their own red blood cells. It is a form of hemolytic anemia, meaning the body can't produce enough blood cells fast…
Prader-Willi syndrome (PWS) is a neurodevelopmental disorder which causes intellectual disability, a deficiency of growth hormones, compulsive behavior, a high risk of obesity, and hyperphagia, a symptom which can be…
There's some good news for the hereditary angioedema patient community this week! Shire, a global biotechnology company focused on rare diseases, announced in the Journal of the American Medical Association (JAMA) publication…
Zealand Pharma A/S (Zealand) recently announced in a press release the start phase 3 trial for short bowel syndrome treatment. The trial investigates the use of glepaglutide as a treatment…
A report on newsmedical.net details a major grant awarded for the research of idiopathic pulmonary fibrosis (IPF). The grant totals $8.9 million, aiming to fund a program for several years.…
A new clinical trial aims to be the first treatment for mitochondrial disease to be granted FDA approval. Whether or not the trial results in a landmark treatment, it is…
Major pharmaceutical company Pfizer recently announced the success of a new experimental product. The drug targets a disease called transthyretin cardiomyopathy, related to amyloidosis, a rare and dangerous disease that affects…
A phase three clinical study is beginning for a new glioblastoma treatment. The new drug is called VAL-083 and is specially designed for treatment of late-stage glioblastoma. The study will…
As parents, we want to protect our children from harm and keep them safe. So when your child is suffering from a rare metabolic disorder, it's natural to feel helpless.…
A Futurism article this week put into very human terms the ongoing question we face when it comes to rare disease treatment and Pharma: What is the cost of life?…
The FDA this week granted orphan drug designation to glepaglutide for the treatment of short bowel syndrome (SBS), developed by Zealand Pharma A/S. SBS is a rare disorder where an…
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