Gene Therapy Trial Begins for Frontotemporal Dementia bit.ly/3NRG9Sp #Dementia #FrontotemporalDementia #FTD

GRIN2B Disorder: Research – A Light on the Path bit.ly/3S766jz #GRIN2B #GRIN2B Disorder #GRINDisorder #RareDisease CureGRIN GRIN2Bsyndrome

Phase 1 Study Begins to Evaluate AGMB-447 for IPF bit.ly/48zJeOM #PulmonaryFibrosis #IPF #IdiopathicPulmonaryFibrosis Pulmonary Fibrosis Foundation

🔬 Chiesi Global Rare Diseases has received FDA approval for Filsuvez, a new topical gel containing birch triterpenes, offering hope to those living with junctional epidermolysis bullosa (JEB) and dystrophic epidermolysis bullosa (DEB). Read more here: buff.ly/3RI8dsC

#RareDisease

Evobrutinib Fails in Phase 3 Trial for Multiple Sclerosis (MS) bit.ly/48Y0EFl #MultipleSclerosis #MS National MS Society

The Telemedicine Revolution: Reshaping Primary Healthcare for Better or Worse? bit.ly/48FSxN9 #Telemedicine #Healthcare

CAR T-Cell Therapy has Revolutionized Treatment for B-Cell Malignancies but Significant Toxicity Still Exists bit.ly/3tNhlEa #BloodCancer #Cancer #Oncology #CARTCellTherapy #Leukemia #Lymphoma

Sarcoidosis: Case Study of Presentation as Isolated Orbital Granuloma bit.ly/47jwiMe #Sarcoidosis #RareDisease #Granuloma FSR

Deucrictibant Reduces Monthly HAE Attacks by 84.5% in Phase 2 Study bit.ly/4aJJXPm #HAE #HereditaryAngioedema #RareDisease US_HAEA

Trial Results Double Survival for Some People with Advanced Bladder Cancer bit.ly/485vEmF #BladderCancer #ClinicalTrial Bladder Cancer Advocacy Network

Parents of Twin Girls with Rett Syndrome Have New Hope for the Future bit.ly/3GSeY68 #RettSyndrome #RareDisease International Rett Syndrome Foundation

Takeda Announces Positive Results for TYK279 to Treat Patients with Active Psoriatic Arthritis bit.ly/4aFkB5o

#PsoriaticArthritis

Family Gets Their Christmas Miracle, Baby Cured of Hyperinsulinism bit.ly/4at8wQn #Hyperinsulinism #ChronicIllness #Christmas #Holidays

Best Christmas Present Ever Was Delivered Surgically bit.ly/3NztgMI #Cholangitis #PrimaryBiliaryCholangitis #PBC #RareDisease #LiverDisease

Rare Classroom: Cutaneous T-Cell Lymphoma bit.ly/41teWLk

#CutaneousTCellLymphoma #Lymphoma #RareClassroom

Rheumatoid Arthritis: Analysis Suggests Efficacy for Combination in Some Patients bit.ly/3TtnkIV #Arthritis #RheumatoidArthritis Arthritis Foundation

Promising Overall Survival Results in Trial for Patients with Gastric Cancer bit.ly/41rUUB4

#GastricCancer

A Girl with Williams Syndrome Gets a Christmas Lights Spectacular bit.ly/3tsM5KH #WilliamsSyndrome #RareDisease #Christmas #Holidays

Drug Combination Shows Promise in Chronic Kidney Disease bit.ly/3TwZJqC #KidneyDisease #CKD #ChronicKidneyDisease National Kidney Foundation

Consider Rare Disease Families at Holiday Time bit.ly/47VM5ln #RareDisease #Holidays #Christmas