Female mammals have XX chromosomes and males have XY. But scientists have never understood how females decide which of their two X's should be active in each of its cells.…
Dr. Harvey Moldofsky Harvey Moldofsky is a professor emeritus at the University of Toronto. His area of focus is SARS, and since the onset of COVID-19, he has been fully…
Ross Douthat, a writer for the New York Times, was first diagnosed with Lyme disease and a Bartonella infection in 2015 from a deer tick bite. The effects have lingered…
Ascendis Pharma has just announced that their investigative therapy for achondroplasia called TransCon CNP has just received Orphan Drug Designation from the European Commission. This same designation has already been…
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…
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…
Parkinson’s UK is nonprofit organization striving to help Parkinson's disease research continue amidst this global pandemic. Their goal is to get clinical research, and specifically studies that necessitate patients participating…
Rare Pediatric Disease Designation has just been granted by the FDA for an investigative therapy for lymphatic malformations (LMs). This therapy is called TARA-002 and was developed by Protara Therapeutics.…
An investigative therapy for hemolytic disease of the fetus and newborn (HDFN) has just received the FDA's Rare Pediatric Disease and Orphan Drug Designations. This therapy is called nipocalimab and…
Moebius Syndrome Moebius syndrome is a rare condition that causes an inability to move the eyes laterally and facial paralysis. A similar type of paralysis can also occur as a…
Fast Track Designation and Rare Pediatric Disease Designation have just been awarded from the FDA to HemoShear Therapeutics. This designation was given to their new investigative therapy for propionic acidemia…
Gaucher Disease Awareness Month takes place every October, but amidst this new global pandemic, planning has taken place early to figure out how to make the month as successful as…
Chemotherapy-induced peripheral neuropathy, or CIPN, is a common side effect of chemotherapy. A recent study aimed to uncover whether or not oxaliplatin chemotherapy increased risk of CIPN for those with…
Janssen Pharmaceuticals has an ongoing Phase 1/2 clinical trial for the inherited retinal disease X-linked retinitis pigmentosa (XLRP). They are investigating an adeno-associated gene therapy called RPGR as a therapeutic…
Scares That Care Scares That Care is a 501(c)(3) organization, first established in 2007 by Joe Ripple, dedicated to fighting the "monsters" of childhood illnesses, breast cancer, and burns. They support…
The EveryLife Foundation has initiated a scholarship fund to help those with rare conditions pursue their academic dreams. These will be one-time awards of 5,000 dollars. Up to 32 patients will…
Duncan DeLooze Mr. DeLooze was diagnosed with hemiplegic migraine, which causes similar symptoms to a stroke. The condition also causes seizures. He's gone through surgeries, back issues, shoulder pain, and chronic…
A study conducted by researchers from the Institute of Cancer Research in London and Cyclacel has recently been published in PLOS ONE. This investigation examined fadraciclib as a potential therapy…
Alpha-synuclein cause damage to mitochondria in patients with Parkinson's disease. These researchers wanted to see if they could see the membrane changes fostered by the alpha-synuclein. The examination of such…
ChemoCentryx has just announced that they have submitted their New Drug Application to the FDA for a novel ANCA-associated vasculitis therapy. It is called avacopan and it's finally something non-steroidal.…
Rare pediatric disease designation has just been granted to Rhythm Pharmaceuticals for their investigative therapy called setmelanotide. This drug is being investigated as a therapeutic option for two conditions- leptin…
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