No healthcare professional wants to hear that a drug being studied in a clinical trial did not meet the endpoints. It means that after countless hours of hard work,…
The FDA has just provided Orphan Drug Designation to Hizentra for the treatment of adults diagnosed with chronic inflammatory demyelinating polyneuropathy (CIPD). This is a maintenance therapy which works to…
Marinus Pharmaceuticals has recently announced updates on its ganaxolone programs for tuberous sclerosis complex (TSC), PCDH19-related epilepsy (PCDH19-RE), and CDKL5 deficiency disorder (CDD). Ganaxolone Ganaxolone is a positive allosteric modulator…
David Fajgenbaum was diagnosed with Castleman disease while he was still a medical student. He was perfectly healthy, but in a few days he had organs failing. He was treated…
A new study has been initiated by a digital health company in the United Kingdom called Congenica Ltd. This study focuses on re-annotating genes that may be associated with…
Galectin Therapeutics has recently started to plan the third phase of its clinical trial of belapectin, which treats nonalcoholic steatohepatitis (NASH). They are making refinements based on a conversation…
According to a story from Targeted Oncology, the results of a recent clinical trial have left drug developers Kite and Gilead with the confidence to proceed with regulatory approval from…
The EMA has just announced that Danicopan has been accepted into its PRIority MEdicines (PRIME) program. Danicopan is being developed for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood condition. PRIME…
According to a story from BioPortfolio, the drug companies bluebird bio, Inc. and the Bristol-Myers Squibb Company have recently announced positive final results for their phase 2 clinical trial. This…
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According to a recently published article in Targeted Oncology, the results of the Phase III CANDOR trial were presented at the 61st ASH annual conference in Orlando, Florida. The…
According to a story from EurekAlert! a recent study that was published in the Journal of Huntington's Disease has concluded that while this rare disease community is excited about what the latest…
Personalized Medicine You may have heard of personalized medicine. It's a new wave of healthcare that aims to provide individualized treatment to patients based on their own genetic makeup. The…
According to a story from Pulmonary Hypertension News, a recent study has determined that cardiopulmonary exercise testing can be an effective method to help identify systemic sclerosis patients that are…
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…
By Danielle Bradshaw from In The Cloud Copy Scientific teams at the Centers for Disease Control (CDC), the National Institute of Environmental Health Sciences (NIEHS), and other research centers have…
According to a story from globenewswire.com, the genetic medicines company Homology Medicines, Inc., has recently released promising early data from its phase 1/2 clinical trial. This clinical trial is testing…
According to a story from PR Newswire, the biopharmaceutical company Soligenix, Inc. has announced recently that its phase 3 clinical trial has officially completed enrollment. This trial is testing the…
Two patients at UMass Memorial Medical Center with Tay-Sachs disease received gene therapy to attempt to correct the mutation in the HEXA gene that causes the disease. The patients that…
According to a story from crweworld.com, the biopharmaceutical company BeiGene, Ltd. recently released the results of a phase 3 clinical trial. The goal of this trial was the comparison of…
According to a story from globenewswire.com, the drug developer Agios Pharmaceuticals, Inc. has recently released a preliminary analysis of phase 2 trial findings that have served to establish proof-of-concept for…
According to a story from Benzinga, the drug company Abeona Therapeutics, Inc. has recently announced plans to move forward with its phase 3 clinical trial following the lifting of the…
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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…
By Caitlin Seida from In The Cloud Copy Although it’s already known that the cause of Alexander disease (AxD), a type of leukodystrophy, is a misfolded protein known as GFAP.…
By Jodee Redmond from In The Cloud Copy University researchers Richard Terek and Qian Chen have published a paper drawing attention to a possible treatment for chondrosarcoma, which is a…
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