Rare Diseases

Monopar Announces Publication of Phase 2 Study Demonstrating ALXN1840 Significantly Improves Copper Balance in Patients with Wilson Disease - For More Information Visit shorturl.at/XfmhN @MonoparTx #Wilson_Disease #Rare_Diseases #Orphan_Drugs #Drug_research

Savara Presents Long-Term IMPALA-2 Data Showing Sustained Efficacy and Safety of Molgramostim in aPAP at ATS 2026 Conference - For More Information Visit shorturl.at/sOouQ @SavaraPharma #Rare_Diseases #aPAP #Autoimmune_Pulmonary_Alveolar_Proteinosis #Orphan_Drugs

CSL Canada Announces First Canadian Patient Receives HEMGENIX® (etranacogene dezaparvovec) Hemophilia B Gene Therapy in Ontario, Canada - For More Information Visit shorturl.at/NsRt5 #HemophiliaB #Rare_Diseases #Orphan_Drugs #HEMGENIX #Drug_Research @CSL

Dyne Therapeutics Initiates Phase 3 FORZETTO Trial of Zeleciment Rostudirsen for Duchenne Muscular Dystrophy (DMD) - For More Information Visit shorturl.at/THW0c @Dyne_tx #Duchenne #Muscular_Dystrophy #DMD #Rare_Diseases #Orphan_Drugs #Pediatric_Diseases

Flow Pharma U.S. Patent Allowed for Broad-Spectrum Ebola Treatment Using Siltuximab and Microsphere Delivery Platform - For More Information Visit shorturl.at/7UdEh #FlowPharma #Rare_Diseases #Ebola

IntraBio Receives Regulatory Authorization to Begin Pivotal Phase III Trial of Levacetylleucine in CACNA1A-Related Disorders Across Participating Regions - For More Information Visit shorturl.at/KjBcJ @IntraBio #CACNA1A_Disorders #spinocerebellar_ataxia_6 #Rare_Diseases

Telomir Reports Peer-Reviewed Publication of Telomir-Zn Data Demonstrating Improved Survival and Multiple Endpoint Benefits in Wilson's Disease - For More Information Visit shorturl.at/8Dl83 @TelomirPharma #Wilson_Disease #Rare_Diseases #Orphan_Drugs #Telomir

A Johns Hopkins Registry Study Found CFTR Modulator Therapies Associated with a 66% Lower Hazard of Death Over Eight Years in Cystic Fibrosis - For More Information Visit shorturl.at/6XM9k #Cystic_Fibrosis #CF #Rare_Diseases #Orphan_Drugs @JohnsHopkinsSPH

Notre Dame Breakthrough Multiple Sclerosis Study Challenges Decades-Old Assumptions and Redefines the Future of Myelin Repair Research - For More Information Visit shorturl.at/0skyE @NotreDame #MS #Multiple_Sclerosis #Rare_Diseases #Orphan_Drugs #Drug_research

New England Journal of Medicine Publishes TETON-1/2 Results Showing Nebulized Tyvaso Significantly Improves Outcomes in Idiopathic Pulmonary Fibrosis - For More Information Visit shorturl.at/nAtHF #NEJM #IPF #Idiopathic_Pulmonary_Fibrosis #Rare_Diseases #Orphan_Drugs

Zenas BioPharma to Present Positive Phase 3 INDIGO Trial Results for Obexelimab in IgG4-Related Disease at EULAR 2026 Congress - For More Information Visit shorturl.at/Av858 #Zenas_BioPharma #EULAR #IgG4_RD #IgG4_Related_Disease #Rare_Diseases #Orphan_Drugs

Cullinan Therapeutics Receives FDA Orphan Drug Designation for CLN-049, a Novel FLT3xCD3 T Cell Engager, in R/R Acute Myeloid Leukemia - For More Information Visit shorturl.at/93Oun @CullinanTx #AML #FDA #Acute_Myeloid_Leukemia #Leukemia #Rare_Diseases

Korro Selects KRRO-111 as Development Candidate for the Potential Treatment of Alpha-1 Antitrypsin Deficiency (AATD) - For More Information Visit shorturl.at/lT908 @KorroBio #Alpha1_Antitrypsin_Deficiency #AATD #Rare_Diseases #Orphan_Drugs #Korro_Bio #Drug_research

Tiziana Life Sciences Announces New Positive Clinical Data for Intranasal Foralumab in Non-Active Secondary Progressive Multiple Sclerosis - For More Information Visit shorturl.at/QoeV0 #Tiziana_Life_Sciences #Multiple_Sclerosis #SPMS #Rare_Diseases #Orphan_Drugs

Egetis to Present Healthcare Resource Drivers and Cost in MCT8 Deficiency at ISPOR 2026 - For More Information Visit shorturl.at/mEeKO #ISPOR @Egetis_EGTX #MCT8_Deficiency #Rare_Diseases #Orphan_Drugs #Drug_research #Allan_Herndon_Dudley_syndrome #AHDS

Amplia Therapeutics Launches First Stage of Registration-Enabling Trial of Narmafotinib in Pancreatic Cancer - For More Information Visit shorturl.at/8PqWK @ampliatx #Pancreatic_Cancer #Rare_Diseases #FDA #Orphan_Drugs #Cancer_Research #Amplia_Therapeutics

FDA Accepts AQNEURSA® sNDA and Grants Priority Review for Ataxia-Telangiectasia in Adults and Pediatric Patients - For More Information Visit shorturl.at/8hyGI @IntraBio #Ataxia_Telangiectasia #Rare_Diseases #FDA #Orphan_Drugs #Drug_research #Pediatric_Disorders

Lantern Pharma Announces Successful Outcome of FDA Type C Meeting Request for HARMONIC™ Phase 2 Trial of LP-300 in Never-Smokers with NSCLC - For More Information Visit shorturl.at/gIeF7 @LanternPharma #FDA #NSCLC #Lung_Cancer #Rare_Diseases #Orphan_Drugs

Alterity Therapeutics Highlights Promising Phase 2 Multiple System Atrophy (MSA) Data and Advances ATH434 Toward Phase 3 Development - For More Information Visit shorturl.at/xjxUD @AlterityT #MSA #FDA #Multiple_System_Atrophy #Rare_Diseases #Orphan_Drugs

Design Therapeutics Reports Positive Phase 1/2 Data for DT-216P2 in Friedreich Ataxia Showing Biomarker and Clinical Improvements - For More Information Visit shorturl.at/XqkHU #Design_Therapeutics #Rare_Diseases #Friedreich_Ataxia #Orphan_Drugs #Drug_research