Cogent Biosciences has just released promising initial data from their Phase 2 clinical trial called APEX. This trial is investigating bezuclastinib, a selective KIT D816V inhibitor, as a treatment for…
Jonathan Rayner works at the University of South Alabama as the Director of the Laboratory of Infectious Diseases. Jonathan has studied ticks since back in 2018. He and his colleagues…
Global Blood Therapeutics (GBT) has just announced that their therapeutics called GBT021601 (GBT601) and inclacumab for sickle cell disease (SCD) have received both Rare Pediatric Disease Designation and Orphan Drug…
Anemia is a common symptom for chronic kidney disease (CKD), but there hasn't been much research done on the association between the two or the adverse outcomes which ensue as…
At the end of May, 2022, the Governor of Connecticut, Ned Lamont, established a permanent Rare Disease Advisory Council (RDAC) for the state by signing House Bill 5500 (HB 5500).…
The FDA has just announced that they have lifted the temporary hold placed on pheNIX gene therapy. This gene therapy was created by Homology Medicine as an investigational treatment for…
The FDA has recently approved combination treatments for those diagnosed with unresectable advanced or metastatic esophageal squamous cell carcinoma. These combination treatments are nivolumab and fluoropyrimidine along with platinum-containing chemotherapy…
Data from a Phase 2 trial examining BI 10115550, an oral treatment for idiopathic pulmonary fibrosis (IPF), has just been released. This data shows that this therapy is able to…
Regeneron Pharmaceuticals Inc. has just released positive results from their Phase 3 investigation of Evkeeza as a treatment for homozygous familial hypercholesterolemia (HOFH). Specifically, this trial studied the treatment for…
CureDuchenne is a nonprofit whose mission is to find a cure for Duchenne muscular dystrophy (DMD), a rare progressive disease characterized by muscular degeneration. It is diagnosed in one individual…
PTC Therapeutics Inc. has just announced that their gene therapy called Upstaza has received a positive opinion from the EMA. After the therapy is ratified by the European Commission (EC),…
Eric Miller was diagnosed with Lyme carditis, which is a very rare complication that can arise from Lyme disease. He is a 43-year-old who works at a boarding school in…
Rhiley Fitch just turned five years old on May 10th. She is diagnosed with a condition called apraxia, which is a rare motor speech disorder. Rhiley is determined to learn…
Action CIND is a charity which strives to raise awareness for chronic immunological and neurological, or CIND, diseases. The organization was founded by Alison Rae, who lives with myalgic encephalomyelitis…
The Bausch + Lomb Corporation has just released new findings from a survey of retina specialists. Their goal was to understand the need for some type of new treatment for…
The FDA has just provided AB001 the Orphan Drug designation for both acute myeloid leukemia (AML) and pancreatic cancer. This small molecule was developed by Vopec Pharmaceuticals and Agastiya Biotech.…
In Detroit, activists recently came together to raise money for pancreatic cancer research through a walk called “The PanCAN PurpleStride walk.” Each year about 62,000 people are newly diagnosed with pancreatic…
Those who are at high risk for pancreatic cancer now have a new screening option thanks to a novel program developed by Hackensack University Medical Center. The Center has launched…
Mereo BioPharma Group has just announced positive results from their Phase 2 study called ASTRAEUS. This study investigated alvelestat (MPH-966), which is an oral neutrophil elastase inhibitor, as a treatment…
Gannex has just announced that they have dosed the first patient in their Phase 2 clinical trial examining ASC42 as a treatment for those diagnosed with primary biliary cholangitis (PBC).…
Hesperos Inc. is a small company based out of Orlando. But, with funding from the NIH NCATS center, they have made exceptional and innovative progress on a novel invention for…
SOTIO Biotech has just announced that the first patient has received the first dose of treatment in their Phase 1/2 trial for SOT102. SOT102 is an investigative treatment for pancreatic…
A recent study published in the American Journal of Gastroenterology has demonstrated that people diagnosed with eosinophilic esophagitis (EoE) are at a higher risk for anxiety, mood disorders, as well as ADHD. With this…
Myotonic dystrophy type 2 is a rare and poorly understood condition. Even less understood is its relation to recurrent ventricular tachycardia, which is even rarer. Myotonic Dystrophy Type 2 Myotonic…
Ala Moshiri from the University of California is both a researcher and an eye doctor. He recently discussed his frustration surrounding the lack of available treatments for so many genetic…
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