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Neurizon Therapeutics Unveils Oral Liquid Formulation of NUZ-001 to Improve ALS Patient Accessibility and Treatment - For More Information Visit shorturl.at/otslD Neurizon #ALS #MND #Rare_diseases #Amyotrophic_Lateral_Sclerosis #OrphanDrugs #Drug_research

Biogen Presents Promising Data on Higher Dose Nusinersen and Long-Term Benefits of Early Spinal Muscular Atrophy (SMA) Treatment - For More Information Visit shorturl.at/XLp5R Biogen #SMA #Rare_Diseases #Spinal_Muscular_Atrophy #OrphanDrugs #Drug_Research

Schrödinger Receives Fast Track Designation for SGR-1505 in the Treatment of Relapsed/Refractory Waldenström Macroglobulinemia - For More Information Visit shorturl.at/oEYoj #Schrödinger #FDA #Rare_diseases #Waldenström_Macroglobulinemia #OrphanDrugs

SineuGene Therapeutics Receives FDA Orphan Drug Designation for SNUG01, a First-in-Class Gene Therapy in the Treatment of ALS - For More Information Visit shorturl.at/ifrYG #SineuGene #ALS #Rare_Diseases #amyotrophic_lateral_sclerosis #FDA #OrphanDrugs

Novadip and Cliniques Universitaires Saint-Luc Report Positive Results for Compassionate Use of NVD003 on Four Pediatric Patients - For More Information Visit shorturl.at/xunU2 #CPT #Rare_diseases #OrphanDrugs #Congenital_Pseudarthrosis_of_the_Tibia #Novadip

FDA Approves Gamifant® (Emapalumab-lzsg) as First-Ever Treatment for Adults and Children with Macrophage Activation Syndrome in Still’s Disease - For More Information Visit shorturl.at/pBZw2 #FDA #Rare_Diseases #Stills_Disease #OrphanDrugs #Drug_Research

argenx Advances ARGX-119 into Registrational Study for Congenital Myasthenic Syndromes Following Positive Phase 1b Results - For More Information Visit checkorphan.short.gy/sFl1eQ argenx #CMS #Rare_Diseases #Congenital_Myasthenic_Syndromes #OrphanDrugs

Biogen Initiates Phase 3 Study of Felzartamab for the Treatment of Primary Membranous Nephropathy - For More Information Visit shorturl.at/O4bo1 Biogen #Primary_Membranous_Nephropathy #PMN #Rare_diseases

Klotho Neurosciences Moves Forward with Manufacturing Gene Therapy for the Treatment of ALS - For More Information Visit shorturl.at/DBIGL Klotho Neurosciences (Nasdaq: KLTO) #amyotrophic_lateral_sclerosis #ALS #Rare_Diseases

Vertex Announces European Commission Approval of ALYFTREK®, a New Once-Daily CFTR Modulator for the Treatment of Cystic Fibrosis - For More Information Visit shorturl.at/Miipp Vertex Pharmaceuticals #Cystic_Fibrosis

Neurogene Announces Registrational Trial Design for Embolden™ Study of NGN-401 Gene Therapy in Rett Syndrome - For More Information Visit shorturl.at/RkpMI Neurogene Inc. #Rett_Syndrome #FDA

Genenta Reports Promising Long-Term Survival and Disease Stabilization in Glioblastoma Patients Treated with Temferon - For More Information Visit shorturl.at/RPuAy Genenta Science (NASDAQ:GNTA) #Glioblastoma #Rare_Diseases #OrphanDrugs #Drug_Research #Genenta

Hemispherian Receives U.S. FDA Orphan Drug Designation for GLIX1 in the Treatment of Malignant Glioma - For More Information Visit shorturl.at/mIRYg #Glioma #Brain_Cancer #FDA #Rare_Diseases

Chemomab Announces Oral Presentation of Positive Nebokitug Phase 2 Clinical Data in Primary Sclerosing Cholangitis at BSG Live’25 - For More Information Visit shorturl.at/W4DNj Chemomab Therapeutics #PSC #Rare_Diseases #Primary_Sclerosing_Cholangitis #OrphanDrugs

AI-Powered Drug Discovery Identifies Vorinostat as Promising Multi-System Treatment for Rett Syndrome - For More Information Visit shorturl.at/eqWY9 Wyss Institute #Rett_Syndrome #Rare_Diseases #OrphanDrugs

Lynozyfic™ (linvoseltamab-gcpt) Receives FDA Accelerated Approval for the Treatment of Relapsed or Refractory Multiple Myeloma - For More Information Visit shorturl.at/eFhmf Regeneron #Rare_Diseases #FDA #MM #Multiple_Myeloma #OrphanDrugs #Drug_Research

New University of Padua ALS Study Reveals How Muscle Stem Cells May Hold the Key to Slowing Disease Progression - For More Information Visit shorturl.at/3kBCV Department of Biology – University of Padova #ALS #MND #Rare_Diseases #Amyotrophic_Lateral_Sclerosis #OrphanDrugs #Medical_Research

Inventiva’s Lanifibranor Shows Promise in Preventing Liver Disease Progression by Reversing Early Vascular Damage in MASH - For More Information Visit shorturl.at/w2vZW #MASH #MASLD #Rare_Diseases

IDMC Provides Positive Recommendation for Bio-Thera Solution’s BAT4406F in Neuromyelitis Optica Spectrum Disorder Based on Interim Analysis of Phase II/III Trial - For More Information Visit shorturl.at/bAGXW Bio-Thera Solutions #Neuromyelitis_Optica #NMOSD #Rare_Diseases

Dizal’s ZEGFROVY® (sunvozertinib) Receives FDA Accelerated Approval as the Only Targeted Oral Treatment for NSCLC with EGFR Exon 20 Insertion Mutations - For More Information Visit shorturl.at/p4zxK Dizal Pharmaceutical #NSCLC #Rare_Diseases #FDA #OrphanDrugs