A Widely Used Antifungal Drug Could be Effective For Treating Congenital Erthropoietic Porphyria
Alexas_Fotos / Pixabay

A Widely Used Antifungal Drug Could be Effective For Treating Congenital Erthropoietic Porphyria

According to a story from Medical Xpress, an international group of scientists from the US, France, and Spain may have discovered that an antifungal agent commonly used to treat skin…

Continue Reading A Widely Used Antifungal Drug Could be Effective For Treating Congenital Erthropoietic Porphyria
Innovative Program in Greenville, SC Helps Patients With Sickle Cell Anemia
StockSnap / Pixabay

Innovative Program in Greenville, SC Helps Patients With Sickle Cell Anemia

According to a story from Greenville Online, the Greenville Health System has begun implementing a comprehensive treatment program for patients with sickle cell disease and its most severe and common…

Continue Reading Innovative Program in Greenville, SC Helps Patients With Sickle Cell Anemia
Experimental Treatment for Anemia Associated With Beta Thalassemia Gets Fast Track Designation
allinonemovie / Pixabay

Experimental Treatment for Anemia Associated With Beta Thalassemia Gets Fast Track Designation

According to a story from PR Newswire, the drug developer Protagonist Therapeutics recently announced that its investigational therapy PTG-300 has earned Fast Track designation from the US Food and Drug…

Continue Reading Experimental Treatment for Anemia Associated With Beta Thalassemia Gets Fast Track Designation
Clinical Trial Data Looking Promising for an Experimental Amyotrophic Lateral Sclerosis Treatment
DarkoStojanovic / Pixabay

Clinical Trial Data Looking Promising for an Experimental Amyotrophic Lateral Sclerosis Treatment

According to a story from BioSpace, the biotechnology company BioElectron recently announced that the results from its Phase 2a clinical trial of its experimental product EPI-589 are looking positive. EPI-589…

Continue Reading Clinical Trial Data Looking Promising for an Experimental Amyotrophic Lateral Sclerosis Treatment

Cambridge Researchers Find That Gene Editing Could Help Treat Mitochondrial Diseases

According to a story from UPI, gene editing technology could be a potential treatment for mitochondrial diseases, a rare, genetic illness that is usually fatal. In a proof-of-concept experiment, researchers…

Continue Reading Cambridge Researchers Find That Gene Editing Could Help Treat Mitochondrial Diseases
Experimental Treatment Shuts Down Hereditary Angioedema Attacks Quickly in Trials
Free-Photos / Pixabay

Experimental Treatment Shuts Down Hereditary Angioedema Attacks Quickly in Trials

According to a story from Angioedema News, an investigational product from BioCryst Pharmaceuticals called BCX7353 was able to quickly subdue swelling attacks in hereditary angioedema in clinical trials. In addition,…

Continue Reading Experimental Treatment Shuts Down Hereditary Angioedema Attacks Quickly in Trials
Experimental Treatment for Peripheral T-Cell Lymphoma Gets Orphan Drug Designation
PDPics / Pixabay

Experimental Treatment for Peripheral T-Cell Lymphoma Gets Orphan Drug Designation

According to a story from globenewswire.com, the pharmaceutical company Portola Pharmaceuticals recently announced that its investigational product cerdulatinib has earned Orphan Drug designation from the US Food and Drug Administration…

Continue Reading Experimental Treatment for Peripheral T-Cell Lymphoma Gets Orphan Drug Designation

Clinical Trial for Duchenne Muscular Dystrophy Gene Therapy Restarts After Hold Lifted

According to a story from statnews.com, the drug developer Sarepta has recently announced the its clinical trial testing an experimental gene therapy has resumed after the FDA lifted the hold.…

Continue Reading Clinical Trial for Duchenne Muscular Dystrophy Gene Therapy Restarts After Hold Lifted

A Boy With Congenital Muscular Dystrophy is Being Supported in a Special Way

Michael Dickerson, aged 42, is originally from Hoedspruit Limpopo, South Africa. Recently, Michael was participating in a series of long distance trail runs when he learned about the story of Daniël…

Continue Reading A Boy With Congenital Muscular Dystrophy is Being Supported in a Special Way
ICYMI: After Rejection, The Familial Chylomicronemia Foundation Urges The FDA to Consider Approving New Treatment
www_slon_pics / Pixabay

ICYMI: After Rejection, The Familial Chylomicronemia Foundation Urges The FDA to Consider Approving New Treatment

According to a story from BioSpace, the U.S. Food and Drug Administration (FDA) recently rejected the approval of the drug Waylivra, which was in development for the treatment of familial…

Continue Reading ICYMI: After Rejection, The Familial Chylomicronemia Foundation Urges The FDA to Consider Approving New Treatment
Clinical Trials For Multiple Myeloma and Other Diseases Fail to Reflect Racial Demographics
Pexels / Pixabay

Clinical Trials For Multiple Myeloma and Other Diseases Fail to Reflect Racial Demographics

According to a story from Mother Jones, patients of black or Native American backgrounds are consistently underrepresented in clinical trials. This occurs even when the trials are for diseases that…

Continue Reading Clinical Trials For Multiple Myeloma and Other Diseases Fail to Reflect Racial Demographics

This New Tech Allows Scientists to Observe The Natural Behavior of Pancreatic Cancer Cells

According to a story from EurekAlert! a cell culture platform developed by scientists associated with Hokkaido University will allow researchers to record and observe the natural behaviors of cancer cells.…

Continue Reading This New Tech Allows Scientists to Observe The Natural Behavior of Pancreatic Cancer Cells
Increasing Collaboration and Partnership is Enhancing The Development of Rare Disease Drugs
rawpixel / Pixabay

Increasing Collaboration and Partnership is Enhancing The Development of Rare Disease Drugs

According to a story from Research & Development, increasing collaboration between a variety of different organizations is helping to propel and improve the develop of new drugs for the treatment…

Continue Reading Increasing Collaboration and Partnership is Enhancing The Development of Rare Disease Drugs