Oral Treatment for Inflammatory Bowel Disease Doses First Patients in Phase 1 Trial
source: pixabay.com

Oral Treatment for Inflammatory Bowel Disease Doses First Patients in Phase 1 Trial

As reported in BioSpace; trials for an oral treatment to treat inflammatory bowel disease (IBD) by Morphic Therapeutics has just dosed their first healthy subjects in the Phase 1 trial.…

Continue Reading Oral Treatment for Inflammatory Bowel Disease Doses First Patients in Phase 1 Trial
Study Identifies Predictive Tools to Shorten Active Surveillance Required in Prostate Cancer
Source: Pixabay

Study Identifies Predictive Tools to Shorten Active Surveillance Required in Prostate Cancer

PracticeUpdate recently published an article relating to a study with the primary goal of lightening the burden that many males experience regarding long-term prostate surveillance. Active surveillance has been preferred…

Continue Reading Study Identifies Predictive Tools to Shorten Active Surveillance Required in Prostate Cancer
Experimental Treatment for Fabry Disease to be Made Available Under Expanded Access
source: pixabay.com

Experimental Treatment for Fabry Disease to be Made Available Under Expanded Access

According to a story from PR Newswire, Chiesi Global Rare Diseases and the biopharmaceutical company Protalix BioTherapeutics, Inc. have recently announced that they have launched an Expanded Access Program (EAP)…

Continue Reading Experimental Treatment for Fabry Disease to be Made Available Under Expanded Access

The aHUS Foundation has Created a Medical Tracker for atypical Hemolytic Uremic Syndrome Patients

aHUS Tracking Atypical hemolytic uremic syndrome (aHUS) is a rare disease that causes blood clots to form within the small blood vessels in the kidneys. These clots lead to organ damage…

Continue Reading The aHUS Foundation has Created a Medical Tracker for atypical Hemolytic Uremic Syndrome Patients
Genomic Testing for Rare Cancers
source: pixabay.com

Genomic Testing for Rare Cancers

According to an article from BioSpace, the TargetCancer Foundation has began enrolling patients in their TRACK study. This study is intended to provide rare cancer patients with individualized treatment options…

Continue Reading Genomic Testing for Rare Cancers
 Pneumonia Has Been Replaced by Sepsis as the Most Common Serious Infection in Vasculitis Hospitalizations
source: pixabay.com

 Pneumonia Has Been Replaced by Sepsis as the Most Common Serious Infection in Vasculitis Hospitalizations

Dr. Jasvinder Singh, a rheumatologist at Alabama’s University in Birmingham is the author of a study that was featured in Arthritis Care and Research. During a recent interview with MedPageToday,…

Continue Reading  Pneumonia Has Been Replaced by Sepsis as the Most Common Serious Infection in Vasculitis Hospitalizations
Results from Two Studies Show Soticlestat Efficacy for CDD and Dup15q Syndrome
https://pixabay.com/en/epilepsy-seizure-stroke-headache-623346/

Results from Two Studies Show Soticlestat Efficacy for CDD and Dup15q Syndrome

  Developmental and epileptic encephalopathies (DEEs), or rare epilepsies, are somewhat of a newer topic. However, researchers are already making strides in creating treatments to address unmet patient needs. On…

Continue Reading Results from Two Studies Show Soticlestat Efficacy for CDD and Dup15q Syndrome
A New Mutation Causing Atypical Hemolytic Uremic Syndrome Has Been Discovered
source: pixabay.com

A New Mutation Causing Atypical Hemolytic Uremic Syndrome Has Been Discovered

Researchers have announced their identification of a specific gene mutation that can lead to atypical hemolytic uremic syndrome (aHUS). This gene is called DGKE and it is not a part of…

Continue Reading A New Mutation Causing Atypical Hemolytic Uremic Syndrome Has Been Discovered

The First Patient has Been Dosed with an Investigative Therapy for Acute Myeloid Leukemia

Immune-One Therapeutics has just announced that they have dosed their first acute myeloid leukemia (AML) patient in their Phase 1 trial investigating IO-202. This therapy is an immune inhibitory receptor that works…

Continue Reading The First Patient has Been Dosed with an Investigative Therapy for Acute Myeloid Leukemia
Possible XSCID Treatment MB-207 Granted Orphan Drug Designation
LionFive / Pixabay

Possible XSCID Treatment MB-207 Granted Orphan Drug Designation

  Just last week, biopharmaceutical company Mustang Bio announced Orphan Drug designation for its therapeutic candidate, MB-207. This unique lentiviral gene therapy is designed for previously treated patients with X-linked…

Continue Reading Possible XSCID Treatment MB-207 Granted Orphan Drug Designation
EC Approves Marketing Authorization of AYVAKYT for Patients with PDGFRA D842V Mutant GIST
Pexels / Pixabay

EC Approves Marketing Authorization of AYVAKYT for Patients with PDGFRA D842V Mutant GIST

  Recently, Blueprint Medicines Corporation ("Blueprint") shared that the European Commission (EC) approved marketing authorization of AYVAKYT (avapritinib). The therapy is designed to treat adult patients with PDGFRA D842V mutated gastrointestinal stromal…

Continue Reading EC Approves Marketing Authorization of AYVAKYT for Patients with PDGFRA D842V Mutant GIST