According to a story from Business Wire, the company CENTOGENE, which is dedicated to researching rare genetic disease, will be partnering with the biotech company Aldeyra Therapeutics, Inc. This partnership…
According to a story from news-medical.net, Mariona Graupera and Frances Viñals, along with Dr. Antoni Riera-Mestre, announced the completion of research that could lead to a new potential treatment for…
According to a story from news-medical.net, the results of a recent research trial demonstrate that the radiation therapy drug AZEDRA (ultratrace iobenguane I131) is an effective and safe treatment option…
Spinal muscular atrophy is a genetic disease with life threatening implications. In order to create effective therapies, doctors need the ability to target the root genetic mutation and stop it.…
A Phase II trial of the experimental treatment SurVaxM, under development by the biotechnology company MimiVax LLC, has produced encouraging interim results for treating patients with newly diagnosed glioblastoma. The…
The Boston-based biopharmaceutical company Berg LLC has announced that their experimental drug candidate BPM 31510 (ubidecarenone) has been awarded Orphan Drug designation by the US Food and Drug Administration (FDA)…
The United States Food and Drug Administration (FDA) has granted the drug tafamidis Breakthrough Therapy designation for treating patients who have transthyretin cardiomyopathy. The full article can be read here,…
According to a story from BioPortfolio, the biopharmaceutical company Acceleron Pharma, Inc., recently announced that the Phase 2 trial for its proprietary, investigational product sotatercept. The medication is in development…
Two organisations, NeuroVive Pharmaceutical AB from Sweden and Yungjin Pharm Corporation based in South Korea, have announced encouraging initial results from a Phase I clinical trial of the experimental medicine…
The companies Centogene and Orphazyme are collaborating on a clinical trial of the experimental small molecule therapy arimoclomol for Gaucher disease. The full article can be read here, at BusinessWire. Gaucher…
A drug called Hemlibra that is currently only approved for treating a subset of patients with haemophilia A has been found to be effective for wider use, reports Hemophilia News…
According to a story from apnews.com, sickle cell disease gets very little research or funding despite that fact that about 100,000 people in the U.S. are affected by it. The…
According to a story from Medical Xpress, chronic fatigue syndrome is one of the most poorly understood diseases. Although the name chronic fatigue syndrome seems to merely suggest that patients…
As of last Monday, the United States Food and Drug Administration (US FDA) its approval of a new drug to treat thrombocytopenia in cases of chronic liver disease (CLD). Keep…
According to a story from Worldpharmanews, a PARP inhibitor drug that is commonly used to treat ovarian cancer for patients with BRCA gene mutations could also be effective against pancreatic…
In order to keep up with the quick-growing field of gene therapies, the United States Food and Drug Administration (FDA) announced it will provide faster methods of approval for new…
The U.S. Food and Drug Administration has granted Breakthrough Therapy designation to the experimental drug Lenti-D, which is being produced by the gene therapy company Bluebird bio to treat cerebral…
Dogs make great pets and companions. They’re often loving and obedient. But could dogs also be the key to curing some forms of cancer? When many people think of dogs…
According to a story from news-medical.net, a recent study has revealed that an ingredient commonly found in toothpaste could help patients with cystic fibrosis. The study was conducted by researchers…
The Melanoma Research Alliance (MRA) and The American Cancer Society (ACS) have announced the first recipients of their new research grants, reports PRNewswire. The grants have been set up as…
According to a story from inhomelandsecurity.com, military veterans are roughly twice as likely to get amyotrophic lateral sclerosis (ALS), a lethal degenerative disease that kills in as little as two…
According to a story from Medical Xpress, the drug adalimumab (marketed as Humira), did not have a noticeable effect on aortic inflammation in patients with psoriatic arthritis or psoriasis. Humira…
According to a story from Cardiovascular Business, The FH Foundation, which is a frontrunning patient advocacy and research organization for familial hypercholesterolemia, recently announced that it has begun enrollment for…
Researchers at Columbia University have found a way to apply CRISPR, a tool used in gene therapies, to new types of disease, reports News Medical. The study was carried out…
A company called Beam Therapeutics is developing a new form of genetic editing, called ‘base editing’, to treat certain genetic diseases. The full story can be read here, at MedCity…
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