-
Mashup Score: 1Diagnosing Primary Immunodeficiency - 2 year(s) ago
Dr. Lisa Forbes Satter provides an overview of Primary Immunodeficiency (PI) and getting a proper diagnosis.
Source: CheckRareCategories: Hem/Onc News and Journals, Latest HeadlinesTweet
-
Mashup Score: 0Cushing’s Disease / Cushing’s Syndrome Research Highlights - 2 year(s) ago
Richard Auchus, MD, PhD Professor at the University of Michigan, discusses the latest clinical research presented at ENDO 2022.
Source: CheckRareCategories: Hem/Onc News and Journals, Latest HeadlinesTweet
-
Mashup Score: 0Advancing Cancer Targeted Therapies - 2 year(s) ago
Dr. Milan Radovich summarizes the company’s comprehensive clinico-genomic database to advance targeted cancer therapy.
Source: CheckRareCategories: Latest Headlines, Rare DiseaseTweet
-
Mashup Score: 0Are Gene Therapy Clinical Trials Safe? - 2 year(s) ago
Dr. Peter Marks of the FDA talks about the safety of clinical trials, including those focused on gene therapy.
Source: CheckRareCategories: Latest Headlines, Rare DiseaseTweet
-
Mashup Score: 0Metachromatic Leukodystrophy Milestones - 2 year(s) ago
Dean Suhr of the MLD Foundation provides an update on the latest clinical and diagnostic advances being made to better manage MLD patients.
Source: CheckRareCategories: Hem/Onc News and Journals, Latest HeadlinesTweet
-
Mashup Score: 0Multiple Sulfatase Deficiency (MSD) Symptoms - 2 year(s) ago
Brenna Brentley, of the United MSD Foundation, explains early symptoms of multiple sulfatase deficiency (MSD) that often leads to a diagnosis.
Source: CheckRareCategories: Hem/Onc News and Journals, Latest HeadlinesTweet
-
Mashup Score: 0Rett Syndrome - 2 year(s) ago
Rett syndrome is a multisystem disorder that primarily affects girls; multiple loss-of-function mutations to the MECP2 gene are the cause of this rare disease.
Source: CheckRareCategories: Hem/Onc News and Journals, Latest HeadlinesTweet
-
Mashup Score: 1Phish Finder and the Undiagnosed Patient - 2 year(s) ago
Dr. Kim Moran discusses how UCB is using phish finder programs to help clinicians find undiagnosed patients.
Source: CheckRareCategories: Hem/Onc News and Journals, Latest HeadlinesTweet
-
Mashup Score: 0The Precision Oncology Alliance - 2 year(s) ago
Dr. Chadi Nabhan summarizes the Caris Precision Oncology Alliance and some of the abstracts the alliance presented at ASCO 2023.
Source: CheckRareCategories: Hem/Onc News and Journals, Latest HeadlinesTweet
-
Mashup Score: 0FDA Approves First Gene Therapy for Duchenne Muscular Dystrophy - 2 year(s) ago
The US FDA has approved the gene therapy, delandistrogene moxeparvovec (Elevidys), to treat patients ages 4 through 5 years with Duchenne muscular dystrophy.
Source: CheckRareCategories: Hem/Onc News and Journals, Latest HeadlinesTweet
Lisa Forbes Satter, MD, Associate Professor of Pediatrics at Baylor College of Medicine, explains why it often takes years to properly diagnose Primary Immunodeficiency #PI #checkrare #rarediseases @WorldPIWeek @ImmunoUK @ImmunoCanada https://t.co/dLJ8IYcU6P