Sarepta Reports Durable Functional Benefits of ELEVIDYS in Older Duchenne Muscular Dystrophy (DMD) Patients at WMS 2026 - For More Information Visit tinyurl.com/3tf9f7yd @Sarepta #Duchenne #DMD #WMS_2026 #Muscular_Dystrophy #Rare_Diseases #Orphan_Drugs

Neurocrine Advances Prader-Willi Syndrome Research and Expands Real-World Evidence for VYKAT XR - For More Information Visit tinyurl.com/2xdtujx4 @neurocrine #Prader_Willi_Syndrome #Rare_Diseases #PWS

Tiziana Life Sciences Files for Orphan Drug Designation with the FDA for Intranasal Foralumab for the Treatment of Multiple System Atrophy - For More Information Visit tinyurl.com/49wwcbhe @TizianaLS #MSA #FDA #Multiple_System_Atrophy #Rare_Diseases #Orphan_Drugs

Satellos Announces Completion of Enrollment in BASECAMP Phase 2 Clinical Trial of Forazapadin for Duchenne Muscular Dystrophy - For More Information Visit tinyurl.com/2s4664jr @SatellosBio #Duchenne #DMD #Muscular_Dystrophy #Rare_Diseases #Orphan_Drugs

Roche’s Fenebrutinib is the First BTK Inhibitor to Receive FDA Filing Acceptance in Both Relapsing and Primary Progressive Multiple Sclerosis - For More Information Visit tinyurl.com/5av9c2zd @Roche #PPMS #FDA #Multiple_Sclerosis #Rare_Diseases #Orphan_Drugs

Johnson & Johnson Reports Two-Year Data Showing Sustained Disease Control With IMAAVY in Adolescents With Generalized Myasthenia Gravis - For More Information Visit tinyurl.com/58hnrh5x @JNJInnovMed #gMG #Rare_Diseases #Orphan_Drugs #IMAAVY #Myasthenia_Gravis

Alyftrek Shows Safety and Improved CFTR Function in Children With Cystic Fibrosis as Young as 2 - For More Information Visit tinyurl.com/2mdnv5v8 @IUMedSchool #Cystic_Fibrosis @VertexPharma #Rare_Diseases

argenx to Present New VYVGART Data in Myasthenia Gravis and CIDP at 2026 AANEM and MGFA Sessions - For More Information Visit tinyurl.com/mvcspwzs @argenxglobal #Myasthenia_Gravis #CIDP #argenx

uniQure Reports Sustained 48-Month Slowing of Huntington’s Disease Progression With Iferzuntirgene Inilparvovec (AMT-130) - For More Information Visit tinyurl.com/yfu6vuca @uniQure_NV #Rare_Diseases #Huntington_Disease #Orphan_Drugs #uniQure #Drug_Research

Egetis Therapeutics Announces U.S. FDA Approval of EMCITATE® (tiratricol) for Patients with MCT8 Deficiency - For More Information Visit tinyurl.com/3384b7su #Egetis_Therapeutics #Rare_Diseases #MCT8_Deficiency #FDA #Allan_erndon_Dudley #Orphan_Drugs

Mirum Pharmaceuticals and Incyte Announce Approval of ATEBRIOZ™ for Adult and Pediatric Patients with Fibrodysplasia Ossificans Progressiva - For More Information Visit tinyurl.com/aj3spxkh @Incyte #FDA @mirumpharma #Rare_Diseases #Orphan_Drugs #Pediatric_Diseases

Single Infusion of CARVYKTI® Delivered Five-Year Treatment-Free Remissions in 50% of Patients in Early Line R/R Multiple Myeloma - For More Information Visit tinyurl.com/3fwz72p7 @JNJInnovMed #Blood_Cancer #Multiple_Myeloma #Rare_Diseases #Orphan_Drugs #CARVYKTI

ORYZON Receives EMA Approval to Initiate the HOPE-2 Phase II Study of Vafidemstat for the Treatment of Phelan-McDermid Syndrome - For More Information Visit tinyurl.com/mvys3nwj #Oryzon_Genomics #EMA #Phelan_McDermid_Syndrome #Rare_Diseases #Orphan_Drugs

Tiziana Life Sciences Announces First Participants Dosed in ALS BANYAN Clinical Trial of Intranasal Foralumab - For More Information Visit tinyurl.com/yx95je94 @TizianaLS #ALS #Rare_Diseases #Amyotrophic_Lateral_Sclerosis #Orphan_Drugs #Lou_Gehrig_Disease

Mirum Pharmaceuticals Announces Primary Endpoint Met in Phase 3 AZURE-1 Study of Brelovitug in Chronic Hepatitis Delta Virus - For More Information Visit tinyurl.com/5748kdck @mirumpharma #Rare_Diseases #HDV #Hepatitis_Delta_Virus #Orphan_Drugs #Brelovitug

European Commission Approves NEZGLYAL® as First Pharmacological Treatment for Childhood Cerebral Adrenoleukodystrophy - For More Information Visit tinyurl.com/3dvykt5s #Minoryx @Neuraxpharm_ES #Neuraxpharm #Adrenoleukodystrophy #cALD #Rare_Diseases

Announces FDA Acceptance and Priority Review of sNDA for Lower Doses of Joenja® To Treat Children With APDS - For More Information Visit tinyurl.com/4w72pjha @PharmingGroupNV #APDS #FDA #Rare_Diseases #Orphan_Drugs #Joenja #Pharming

Bristol Myers Squibb’s ZENBEXUS Shows Significant Improvement in MRD-Negative Complete Response in R/R Multiple Myeloma - For More Information Visit tinyurl.com/yc7jbnyp @bmsnews #Multiple_Myeloma #Blood_Cancer #RRMM #Rare_Diseases #Orphan_Drugs

SL Science Unveils New Preclinical Data Demonstrating Potent γδ T Cell Activity Against Chemotherapy-Resistant Glioblastoma - For More Information Visit tinyurl.com/45e5u46m @SLScience_SLBT #Glioblastoma #Rare_Diseases #Orphan_Drugs #Brain_Cancer

Children With Cystic Fibrosis on Kaftrio Maintain Lung Function After Reducing Airway-Clearance Treatments - For More Information Visit tinyurl.com/mu87x6b6 @VertexPharma #Cystic_Fibrosis #Kaftrio #Rare_Diseases #Orphan_Drugs #Lung_Diseases #Pediatric_Diseases