Johnson & Johnson Reports 77% Overall Response Rate with Subcutaneous Amivantamab Combination in First-Line Head and Neck Cancer Study - For More Information Visit tinyurl.com/5bu6z6sr @JNJInnovMed #HNSCC #Head_and_Neck_Cancer #Rare_Diseases #Orphan_Drugs

Mirum Pharmaceuticals Reports Positive PROGRESS Study Results for ATEBRIOZ in Children Aged 6 to Under 12 With Fibrodysplasia Ossificans Progressiva - For More Information Visit tinyurl.com/4mbh4xfk @mirumpharma #FOP #Fibrodysplasia_Ossificans_Progressiva #Rare_Diseases

Solid Biosciences Announces European Commission Orphan Designation for SGT-212 and Continued Progress in the FALCON Trial in Friedreich’s Ataxia - For More Information Visit tinyurl.com/58j493es @Solid_Bio #Friedreich_Ataxia #Rare_Diseases #Orphan_Drugs #Brain_Diserders

TransCon® PTH (Palopegteriparatide) Sustains Clinically Meaningful Kidney Function Improvements in Adults with Hypoparathyroidism for Up to 5 Years - For More Information Visit tinyurl.com/596zdbcu @AscendisPharma #Hypoparathyroidism #Rare_Diseases #Orphan_Drugs

Nuvectis Reports Complete and Durable Responses in BRAF V600-Mutated Recurrent or Progressive Gliomas in NXP200 Phase 1b Study - For More Information Visit tinyurl.com/4xxnv8e6 @Nuvectis #Rare_Diseases #Gliomas #Orphan_Drugs #NXP200 #Brain_Cancers

Ris-Rez Reduced Risk of Disease Progression or Death by 67% Versus Chemotherapy in Patients with Relapsed Osteosarcoma - For More Information Visit tinyurl.com/45ah6tb2 @GSK #Osteosarcoma #Rare_Diseases #Orphan_Drugs #Bone_Cancer #Cancer_Research #Ris_Rez

Regeneron Reports Long-Term Phase 3 Data Showing Sustained Survival Benefits With Libtayo in High-Risk Cutaneous Squamous Cell Carcinoma - For More Information Visit tinyurl.com/32yvnhv4 @Regeneron #CSCC #Squamous_Cell_Carcinoma #Rare_Diseases #Orphan_Drugs #Libtayo

Novartis’ Remibrutinib Delivers Comprehensive Disease Control in Phase III RMS Trials, with a Robust Safety Profile - For More Information Visit tinyurl.com/4w3syjas #RMS #Multiple_Sclerosis #Rare_Diseases #EMA #FDA #Orphan_Drugs @Novartis #Remibrutinib

Genmab’s Rina-S Demonstrates Significant Progression-Free and Overall Survival Benefits in Phase 3 Trial for Platinum-Resistant Ovarian Cancer - For More Information Visit tinyurl.com/2hmmw37c @Genmab #Ovarian_Cancer #Rare_Diseases #Orphan_Drugs #Cancer_research

Roche Presents Interim Phase III Data Showing Sefaxersen Significantly Reduced Proteinuria in People with IgA Nephropathy (IgAN) - For More Information Visit tinyurl.com/ycxnubr5 @Roche #Rare_Diseases #IgAN #Orphan_Drugs #IgA_Nephropathy #kidney_disease

Neurocrine Biosciences Publishes Expert Perspectives on Comprehensive Assessment and Management of Hyperphagia in Prader-Willi Syndrome - For More Information Visit tinyurl.com/ysms8vax @neurocrine #Prader_Willi_Syndrome #Rare_Diseases #Orphan_Drugs #Hyperphagia

Korro Reports Best-in-Class Preclinical Data for KRRO-111 showing Potential Benefits for Lung and Liver Symptoms of Alpha-1 Antitrypsin Deficiency - For More Information Visit tinyurl.com/5y82e65n @KorroBio #AATD #Alpha_1_Antitrypsin_Deficiency #Rare_Diseases

BioNTech and Gilead Team Up to Advance Promising Pumitamig and Trodelvy Combination in Phase 3 Trial for Triple-Negative Breast Cancer - For More Information Visit tinyurl.com/y49p85rh @BioNTech_Group #TNBC @GileadSciences #Breast_Cancer #Rare_Diseases

Prilenia and Ferrer Announce Authorization for EU Initiation of Confirmatory PRECISE-HD Study of Pridopidine in Huntington's Disease - For More Information Visit tinyurl.com/bdzxfy7h @PrileniaTx @ferrerforgood #Huntington_Disease #Rare_Diseases #Orphan_Drugs

Efgartigimod Improves Mobility and Daily Function in Young Woman With Refractory LEMS After Multiple Prior Treatments - For More Information Visit tinyurl.com/5n6kv3jd @argenxglobal #LEMS #Rare_Diseases #Orphan_Drugs #Lambert_Eaton_Myasthenic_Syndrome

Savara Reports Molgramostim Improvements in Gas Transfer, Respiratory Quality of Life, and Surfactant Burden in aPAP Patients - For More Information Visit checkorphan.org/news/514703/ @SavaraPharma #aPAP #Autoimmune_Pulmonary_Alveolar_Proteinosis #Rare_Diseases

Opus Genetics Receives FDA Clearance for OPGx-RDH12 Gene Therapy to Treat Rare RDH12-Associated Leber Congenital Amaurosis - For More Information Visit tinyurl.com/ycx2yzwt #Opus_Genetics #RDH12_LCA #FDA #Leber_Congenital_Amaurosis #Rare_Diseases #Orphan_Drugs

European Commission Approves KEYTRUDA® Plus Padcev® as Perioperative Treatment for Adults With Resectable Muscle-Invasive Bladder Cancer - For More Information Visit tinyurl.com/3phcazz4 @Merck #MIBC #Bladder_Cancer #KEYTRUDA #Rare_Diseases #Orphan_Drugs

Dartmouth Study Links Indoor Fine-Particle Exposure to Changes in Tissue-Repair Signaling in Adults With Cystic Fibrosis - For More Information Visit tinyurl.com/seukdcj3 @DHMCandClinics #Cystic_Fibrosis #CF #Dartmouth #Rare_Diseases #Orphan_Drugs #Lung_Diseases

Neurogene Announces Upcoming Presentations Highlighting Key Differentiators of NGN-401 for Rett Syndrome at the ESGCT Congress - For More Information Visit tinyurl.com/yn36d5sk @NeurogeneInc #ESGCT #Rett_Syndrome #Rare_Diseases #Orphan_Drugs #Brain_Disorders