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Genetic Disorders

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Notizia - Recent Pharma, Healthcare and Biotech Happenings

FDA Approves Vertex’s ALYFTREK for Cystic Fibrosis; ZEPBOUND Gets FDA Approval for Obstructive Sleep Apnea in Obese Adults; Tonix’s TNX-102 SL NDA Accepted for Fibromyalgia; Quoin’s QRX003 Clears FDA for Netherton Syndrome Study; FDA Approves First Mesenchymal Stromal Cell Therapy for Acute GvHD

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FDA Grants Fast Track for Lin BioScience’s LBS-007; Alnylam’s AMVUTTRA sNDA Under Review; FDA Approves RAPIBLYK for Atrial Fibrillation; Applied Therapeutics Receives CRL for Govorestat in Galactosemia; R289 Gets Fast Track for Lower-Risk MDS.

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5 Promising Pompe Disease Drugs in Early-stage Development

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5 Promising Exosome-based Therapies Paving the Way for Personalized Medicine

Feature-Transforming Pompe Disease Care_ Pioneering Innovations and Strategic Advances in Treatment

Transforming Pompe Disease Care: Latest Innovations and Strategic Advances in Treatment

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Exploring Growth Opportunities in the Hypophosphatasia Treatment Market: Navigating the Ultra-rare Disease Landscape

Duchenne Muscular Dystrophy Market

Duchenne Muscular Dystrophy Market: What’s More Beyond Exon-Skipping Therapies?

Duchenne Muscular Dystrophy Advances Challenges and the Future of Treatment

Duchenne Muscular Dystrophy: Advances, Challenges, and the Future of Treatment

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Gene therapy: Following Pfizer’s Unexpected Failure, Sarepta is celebrating its much-awaited full approval! Sarepta’s stock price has shot up over 32%

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Takeda Showcase Phase III Results for Soticlestat; Imfinzi and Chemotherapy Combination Gains US Approval; Nipocalimab Shows Notable Efficacy in Phase II Study; Bristol Myers Squibb’s Augtyro FDA Approval; AstraZeneca’s Farxiga FDA Approval

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X4 Pharmaceuticals’ XOLREMDI FDA Approval; ONO to Acquire Deciphera Pharmaceuticals; Johnson & Johnson’s SIRTURO CHMP Approval; BeiGene’s Tislelizumab EC Approval; Sanofi’s Rilzabrutinib LUNA 3 Phase III Trial

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Breakthroughs in Alport Syndrome Treatment: A New Era of Hope

Navigating the Healthcare Horizon: Odyssey of Mergers, Funding, and Acquisitions in 2024

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Unleashing the Power of mRNA: A Revolutionary Approach to Vaccines and Therapeutics

Pharma News for Merck, BMS, Sanofi, Juvena

Merck’s KEYTRUDA as Adjuvant Therapy for RCC Patients; BMS Receives Positive CHMP Opinion for CAR T Cell Therapy Abecma for Multiple Myeloma; FDA Approves Dupixent for Eosinophilic Esophagitis; Juvena Receives FDA Orphan Drug Designation for JUV-161; European Commission Authorizes GSK’s Omjjara; ENHERTU Granted Priority Review in the US for for metastatic HER2-positive Solid Tumors

Pharma News for Exelixis, AstraZeneca, Kyverna

BMS, and Exelixis’s Opdivo + CABOMETYX in First-Line Advanced Renal Cell Carcinoma; AIRSUPRA Now Available as the First and Only FDA-approved Anti-inflammatory Rescue Option for Asthma; AstraZeneca’s Voydeya Receives First-ever Regulatory Approval; EMA Grants ODD to GC Biopharma’s Sanfilippo Syndrome (Type A) Treatment; FDA Approves NRx Pharma’s IND Application of NRX-101; FDA Fast Track Designation to Kyverna’s KYV-101

emerging-therapies-for-achondroplasia-treatment

Achondroplasia Treatment Space: Hunt for Potential Curative Therapies

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