Rare Diseases

Nouscom Selected for Oral Presentation on NOUS-209 Re-treatment and Long-Term Follow-Up in Lynch Syndrome Carriers at ESMO Congress 2026 - For More Information Visit shorturl.at/58shg #Nouscom #ESMO #Lynch_Syndrome #Rare_Diseases #Orphan_Drugs #Cancer_research

Akagera Medicines Reports Phase 1 Data Support AKG-100 as a Potential Long-Acting Therapy for Drug-Resistant Tuberculosis - For More Information Visit shorturl.at/qZkfl #Akagera_Medicines #Tuberculosis

Oncolytics Biotech® Receives Fast Track Designation for Pelareorep in Second-Line and Later Squamous Cell Carcinoma of the Anal Canal - For More Information Visit tinyurl.com/mr3rjc9x @Oncolytics #Rare_Diseases #SCAC #Orphan_Drugs #Anal_Cancer #Cancer_research

FAST Awards Grant to Nobel Laureate Frances Arnold to Accelerate Next-Generation Therapeutic Tools for Angelman Syndrome - For More Information Visit shorturl.at/5lJRa @cureangelman #Angelman_Syndrome

Dyne Therapeutics Announces FDA Acceptance of Biologics License Application (BLA) for Z-Rostudirsen in Exon 51 Duchenne Muscular Dystrophy - For More Information Visit shorturl.at/WZ09D @Dyne_tx #DMD #Duchenne #Muscular_Dystrophy #Rare_Diseases #Orphan_Drugs #FDA

Nuvation Bio Reports Positive Long-term Phase 2 Safusidenib Data and Launches New Phase 3 And Phase 2 Trials for IDH1-Mutant Glioma - For More Information Visit shorturl.at/BuGqU @nuvationbioinc #Glioma #Brain_Cancers #Rare_Diseases #Orphan_Drugs

Scholar Rock Says EMA Awaits FDA Decision on Catalent Indiana, Potentially Delaying Apitegromab SMA Approval Opinion Until Later 2026 - For More Information Visit shorturl.at/IsiZP @ScholarRock #EMA #FDA #Spinal_Muscular_Atrophy #Rare_Diseases #Orphan_Drugs

HEALEY ALS Platform Trial Regimen I Enrollment Completed – Topline Results for NUZ-001 Accelerated to Q2 CY2027 - For More Information Visit shorturl.at/f5loi @Neurizon_ltd #amyotrophic_lateral_sclerosis #ALS #Rare_Diseases #Lou_gehrig_Disease #Orphan_Drugs

FDA Grants Traditional Approval to Novartis' Fabhalta to Slow Kidney Function Decline in Adults with Primary IgA Nephropathy (IgAN) - For More Information Visit shorturl.at/3rgGV @Novartis #IgA_Nephropathy #IgAN #Rare_Diseases #Orphan_Drugs #Kidney_Diseases

Spur Therapeutics Doses First Patient in Phase 3 GALILEO-3 Trial of FLT201 Gene Therapy for Gaucher Disease Type 1 - For More Information Visit shorturl.at/z5H1c #Spur_Therapeutics #Gaucher_Disease #GD1

UT Tyler Study Shows How CFTR Modulators Reduce P. aeruginosa Burden but Leave Persistent Bacterial Aggregates in Cystic Fibrosis - For More Information Visit shorturl.at/gTug3 @UTTyler #Cystic_Fibrosis #Rare_Diseases #Orphan_Drugs #Lung_Diseases #Lung_Disorders

AlphaRose Therapeutics Announces “N-of-1” Drug Repurposing Platform for Rare Sanfilippo Syndrome Type B - For More Information Visit shorturl.at/dKG0O @alpharoseco #AlphaRose_Therapeutics #MPS_IIIB #Sanfilippo_Syndrome #Rare_Diseases #Orphan_Drugs

Insmed Reports Positive 12-Month Phase 2b Extension Data for Once-Daily TPIP in Pulmonary Arterial Hypertension, Supporting Phase 3 Development - For More Information Visit shorturl.at/4ZuzJ @Insmed #PAH #Pulmonary_Arterial_Hypertension #Rare_Diseases #Orphan_Drugs

aTyr Pharma to Present Subgroup Analysis of Phase 3 EFZO-FIT™ Study of Efzofitimod in Pulmonary Sarcoidosis at WASOG 2026 - For More Information Visit shorturl.at/kJ1o8 @aTyrNews #Rare_Diseases #WASOG #Pulmonary_Sarcoidosis #Orphan_Drugs #Lung_Diseases

US Food and Drug Administration Grants Priority Review to Roche’s Gazyva/Gazyvaro for Adults with Primary Membranous Nephropathy (pMN) - For More Information Visit shorturl.at/yDhUS @Roche #FDA #pMN #Rare_Diseases #Orphan_Drugs #Primary_Membranous_Nephropathy

Hemab Therapeutics Presents Clinical and Preclinical Data from Sutacimig in Glanzmann Thrombasthenia and Factor VII Deficiency at the ISTH 2026 Congress - For More Information Visit shorturl.at/H7Coi @Hemab_Tx #Glanzmann_Thrombasthenia #Factor_VII_Deficiency

Atrium Therapeutics Announces FDA Clearance of Investigational New Drug Application for ATR 1072 for Treatment of PRKAG2 Syndrome - For More Information Visit shorturl.at/3QlsM #Atrium_Therapeutics #FDA #PRKAG2_Syndrome #Rare_Diseases #Orphan_Drugs #Drug_Research

FDA Grants Fast Track Designation to SOTIO’s SOT109, a CDH17-targeting ADC for Colorectal Cancer Patients who Have Exhausted Standard Treatment Options - For More Information Visit shorturl.at/NLrWe @SOTIO #FDA #Colorectal_Cancer #rare_Diseases #Orphan_Drugs

Skyhawk Therapeutics Announces Expansion of its Pivotal FALCON-HD Clinical Trial for SKY-0515 in Huntington’s Disease to the US, Canada and the UK - For More Information Visit shorturl.at/gNMyw #Orphan_Drugs #Huntington_Disease #Rare_Diseases #Skyhawk_Therapeutics

Dompé Doses First Patient in Phase 3 Study of Cenegermin-bkbj in Non-Arteritic Anterior Ischemic Neuropathy (NAION) - For More Information Visit shorturl.at/8VZir #Dompé #NAION #Rare_diseases