Mashup Score:8
CheckRarePKU Treatment: Medical Foods vs Kuvan vs Palynziq - 5 days
Mashup Score:8
Twitter Feed: CheckRare
Mashup Score:6
CheckRareDravet Syndrome Clinical Trial With STK-001 Continues - 5 days
Mashup Score:6
Barry S. Ticho, MD, PhD, Chief Medical Officer at Stoke Therapeutics, discusses the phase 1/2a MONARCH study, which is evaluating STK-001 in patients with...
-
Barry S. Ticho, MD, PhD, Chief Medical Officer at Stoke Therapeutics, discusses the phase 1/2a MONARCH study evaluating STK-001 in patients with Dravet syndrome. https://t.co/Y8K9aJW7Jj @StokeTx @curedravet @DSFeu @DravetUK @NIH #DravetSyndrome #StokeTherapeutics #raredisease - view on twitter
Mashup Score:5
CheckRareMyasthenia Gravis : A History Lesson - 3 days
Mashup Score:5
Dr. Henry Kaminski, of George Washington University, provides a brief history lesson on our understanding of the pathophysiology of myasthenia gravis...
-
Henry Kaminski, MD, Professor of Medicine at The George Washington University, provides a brief history lesson on our understanding of myasthenia gravis #MG. @GWtweets @MGNet__ @MyastheniaOrg #checkrare #raredisease #myastheniagravis https://t.co/ynEwKJnCZB - view on twitter
Mashup Score:4
CheckRareUsing Cryptocurrency To Develop Orphan Drugs for Rare Diseases - 2 days
Mashup Score:4
Alok Tayi, PhD, Founder and CEO of Vibe Bio, gives an overview of his company, its mission and objectives, and how it plans to...
-
Alok Tayi, Ph.D., Founder and CEO of Vibe Bio, gives an overview of his company, its mission and objectives, and how it plans to advance the development of rare disease therapies. @VibeBio @Chelseashopela1 https://t.co/1xZnLUxmhq #VibeBio #Lafora #ChelseasHope #rarediseases - view on twitter
Mashup Score:3
CheckRarePAH Research Highlights: CHEST 2022 - 6 days
Mashup Score:3
Jean Elwing, MD, of the University of Cincinnati College of Medicine provides an overview of the latest clinical research about PAH presented at CHEST...
-
Dr. Jean Elwing of the @UCincyMedicine overviews the latest clinical research about #PAH presented at CHEST 2022. PAH is a rare, progressive disorder characterized by high blood pressure in the pulmonary arteries. @LDRTC_USA https://t.co/JSw7jnCl2a #rarediseases #CME https://t.co/JSw7jnCl2a - view on twitter
Mashup Score:3
CheckRareWhat Is Mastocytosis? - 5 days
Mashup Score:3
Dr. Mariana C. Castells, Professor of Medicine at Harvard Medical School, gives an overview of...
-
Mariana C. Castells, MD, PhD, Division of Allergy and Clinical Immunology, Brigham and Women’s Hospital, and Professor of Medicine, Harvard Medical School, gives an overview of mastocytosis. @BrighamWomens @BrighamDGIM @harvardmed @MastocytosisCA https://t.co/53MnZPWpNd - view on twitter
Mashup Score:3
CheckRarePlans for Two Phase 3 Studies Testing Oxypurinol To Treat ADPKD Announced - 4 days
Mashup Score:3
Dr. Allen Davidoff, CEO of XORTX Therapeutics, discusses two phase 3 studies set to launch in the next two years evaluating oxypurinol in patients...
-
Allen Davidoff, PhD, CEO of XORTX Therapeutics, discusses two phase 3 studies set to launch in the next two years evaluating oxypurinol in patients with autosomal dominant polycystic kidney disease #ADPKD @XORTXinc #checkrare #rarediseases #XORTX https://t.co/WcCkqgoQKG - view on twitter
Mashup Score:3
CheckRareAdvances in Gene Therapy for Lysosomal Diseases - 6 days
Mashup Score:3
Drs Goker-Alpan and Jodele discuss current trends in gene therapy for lysosomal storage...
-
CME program: Ozlem Goker-Alpan, MD, at the LDRTC and Sonata Jodele , MD, at Cincinnati Children’s Hospital Medical Center discuss the current trends in gene therapy for lysosomal storage diseases and some safety concerns @LDRTC_USA @CincyChildrens https://t.co/78g6dh4pN0 - view on twitter
Mashup Score:2
CheckRareWhat Is Autosomal Dominant Polycystic Kidney Disease (ADPKD)? - 5 days
Mashup Score:2
Dr. Allen Davidoff, CEO of XORTX Therapeutics, gives an overview of autosomal dominant polycystic kidney disease...
-
Allen Davidoff, Ph.D., CEO of XORTX Therapeutics, gives an overview of autosomal dominant polycystic kidney disease #ADPKD @XORTXinc #checkrare #raredisease https://t.co/6rUqGAHy6K - view on twitter
Mashup Score:2
CheckRareGH Deficiency Research Highlights - 5 days
Mashup Score:2
Paul Saenger, MD MACE, Professor of Pediatrics Emeritus at Albert Einstein College of Medicine, provides an overview of the latest clinical research presented at...
-
New CME Program: Paul Saenger, MD MACE, Professor of Pediatrics Emeritus at the Albert Einstein College of Medicine, discusses the latest clinical research about Growth Hormone Deficiency (GHD). https://t.co/OJO6vZeLq2 @EinsteinMed @AwarenessGhd @HumanGrowth_Fdn #RareDisease https://t.co/OJO6vZeLq2 - view on twitter
More coverage:
#raredisease
Dr. Jerry Vockley, of UPMC Children’s Hospital of Pittsburgh, discusses the limitations of the current treatment options for phenylketonuria...
Gerald Vockley, MD, PhD, Head of the Division of Medical Genetics at UPMC Children’s Hospital of Pittsburgh, discusses new data testing a probiotic to treat individuals with phenylketonuria. #PKU #raredisease @ChildrensPgh @howmuchphe @official_espku https://t.co/7u3PzI0Rby - view on twitter