Corbus Pharmaceuticals has just announced that participant enrollment for their Phase 3 trial of lenabasum for dermatomyositis has been completed. Dermatomyositis Dermatomyositis (DM) is a rare autoimmune disease. It can…
The Cystinosis Research Network (CRN) has created a new program to bring light to cystinosis families during these uncertain times. It is called the Brighter Days Care Package Program. Brighter…
Would you be shocked to find out that before Evrysdi, there were zero orally-administered drugs approved to treat patients with spinal muscular atrophy (SMA)? But now, things have changed. Last week,…
Nobody knows the exact cause of Parkinson's disease. However, many researchers believe that environmental triggers may play a factor. Now, the Michael J. Fox Foundation (MJFF) is encouraging American…
According to a story from GlobeNewswire, the drug development company Corbus Pharmaceuticals Holdings, Inc., has recently announced that it has completed its enrollment of patients for an upcoming phase 3…
A new study has found a new way to measure memory in both animals and people. The hope is that this assessment will aid in the research of Parkinson's disease and…
We have learned a lot, but still have much to learn about COVID-19. Researchers have seen a link between COVID-19 and chronic fatigue syndrome (myalgic encephalomyelitis) as well as Guillain-Barre syndrome, which…
Arterial Dissection Arterial dissections refer to the tearing of the inner wall of an artery. Pregnancy creates stress on the body's blood vessels and in rare cases dissections can occur.…
Researchers have been trying for years to better understand the mutations which cause the rare disorder called Charcot-Marie-Tooth disease (CMT). Tremendous progress has been made. But now that we know…
As reported by the National Hemophilia Foundation, researchers are seeking to understand what patients with chronic pain need and want, so as to make treatment safer and more effective. In…
Within six months of receiving funding from the Chan Zuckerberg Initiative (CZI), the TANGO2 Research Foundation (T2RF) is proud to announce funding of five new research projects and the hiring…
Alnylam Pharmaceuticals has submitted their clinical trial authorization (CTA) application to the Medicines and Healthcare Products Regulatory Agency (MHRA) for their RNAi therapy, ALN-HSD, a treatment for nonalcoholic steatohepatitis (NASH).…
According to Ryan McDonald of Cure Today, the FDA recently granted Breakthrough Therapy Designation to MK-6482, an investigational HIF-2α inhibitor developed by Merck. The orally-administered drug, designed to treat…
Rare disease or health-related foundations often encourage patient advocacy, research, and discussion around new treatments. According to Parkinson's News Today, the Parkinson's Foundation is doing their part in advancing…
Earlier this week, biotechnology company Time Technologies announced that its drug candidate SM-88 received Orphan Drug Designation from the FDA for the treatment of pancreatic cancer. The FDA defines…
A recent article in The American Journal of Managed Care (AJMC) heralds the approval of a third CAR T-cell therapy, called Tecartus. Kite Pharma’s cell-based gene therapy is the first…
According to a story from GlobeNewswire, the biopharmaceutical company Ovid Therapeutics Inc. and Columbia University have recently entered a collaborative partnership that will be focused on developing gene-based treatments for…
According to a story from GlobeNewswire, the first patient has been dosed in a recent clinical trial testing the experimental therapy tasquinimod as a treatment for multiple myeloma, a rare…
According to a story from BioSpace, the biotechnology company Tyme, Inc. has just announced that it has earned Orphan Drug designation for its experimental product candidate SM-88. SM-88, also known…
A recent article in The American Journal of Managed Care (AJMC) heralds the approval of a third CAR T-cell therapy, called Tecartus. Kite Pharma’s cell-based gene therapy is the first…
August is recognized as Spinal Muscular Atrophy Awareness Month. The goal of this event is to help spread information about this rare disorder among the general public and the broader…
According to a story from the CureDuchenne Blog, Dr. Kevin Flanigan of Nationwide Children's Hospital has recently dosed two Duchenne muscular dystrophy patients with an experimental gene therapy. This gene…
According to a story from GlobeNewswire, the biopharmaceutical company GW Pharmaceuticals plc recently announced that its CBD oral solution Epidiolex® has recently been approved as a treatment for seizures associated…
According to a story from mlive.com, the state of Michigan has confirmed its first case of acute flaccid myelitis, a rare disease that tends to affect children, this year. There…
In 1986, Judy and Larry Pichon were married. The couple lived a happy life in Louisiana, though their days were sometimes complicated by Judy's health. She had granulomatosis with…
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