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Genetic Disorders

Notizia - Recent Pharma, Healthcare and Biotech Happenings

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

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Pharma News for Cytokinetics, Chiesi, AstraZeneca, Zevra

Cytokinetics Announces Results From SEQUOIA-HCM Clinical Trial of Aficamten; FDA Approves Chiesi’s FILSUVEZ Topical Gel; Bristol Myers Squibb Adds Premier Radiopharmaceutical Platform; AstraZeneca to Acquire Gracell; Zevra Therapeutics Announces Resubmission of Arimoclomol New Drug Application; Innovent Dosed First Participant in Phase 3 Clinical Study (GLORY-2) of Mazdutide (IBI362)

expanding-market-of-complement-inhibitor

The Expanding Market of Complement Inhibitors

Pharma News for Merck, Ipsen, bluebird bio

Merck and Moderna Initiate Study to Evaluate V940; FDA Approves Vertex and CRISPR Therapeutics’ CASGEVY for SCD; Novartis Updated on its Investigational Iptacopan Phase III Study; FDA Grants Priority Review for New Drug Application for Elafibranor; FDA Approves bluebird bio’s LYFGENIA for Patients SCD; FDA Fast Track Designation for DMD Gene Therapy

Pharma News for AskBio, Almirall, Bayer, Pfizer

Key Updates on Phase 1 Trial of AB-1005 Gene Therapy for Multiple System Atrophy-Parkinsonian Type; European Commission Approves EBGLYSS; Bayer Stopped OCEANIC-AF Study; Pfizer and Astellas’ XTANDI Approved by FDA; FDA Orphan Drug Designation to Epic Bio’s EPI-321; FDA Fast Track Designation to Chemomab’s CM-101 for PSC

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Emerging Therapies for Niemann-Pick Disease Type C Treatment: The Road to Progress

Molecular Modeling Market Analysis

Revolutionizing Drug Discovery and Beyond: The Power of Molecular Modeling

aav-gene-therapies-for-hemophilia-b-treatment

AAV Gene Therapies for Hemophilia B Treatment: The Road to a Cure

primary-hyperoxaluria-treatment

Can Novo’s Rivfloza Outperform Its Rival Alnylam’s Oxlumo in Primary Hyperoxaluria Treatment Space?

netherton-syndrome-treatment

Netherton Syndrome: Unraveling the Uncertainties of a Rare Genetic Skin Disorder

orchards-otl-200-for-metachromatic-leukodystrophy-treatment

Orchard’s OTL-200 to Enter US Metachromatic Leukodystrophy Treatment Space After EU

huntingtons-disease-treatment

A Turning Point: INGREZZA’s Impact on Huntington’s Disease Treatment and the Rise of Novel Therapies

Pharma News for Faron, Roche, Ergomed, Menarini

Daiichi Sankyo’s Trastuzumab Deruxtecan; ODD to Bexmarilimab for AML; Roche’ Alecensa; Ergomed Aims To Go Private; Tagraxofusp Receives ODD in Japan for BPCDN; FDA Fast Track Designation to Abliva’s KL1333

fabry-disease-treatment

Fabry Disease – A Market Perspective On The Emerging Pipeline

homozygous-familial-hypercholesterolemia-treatment

Labyrinth of LDL-C: Navigating Challenges in Homozygous Familial Hypercholesterolemia Treatment

Pharma News for Gilead, Incannex, Avidity

Eylea HD Injection 8 Mg Approved By FDA; Veopoz Receives FDA Approval for CHAPLE Disease Treatment; FDA Places Second Partial Clinical Hold on AML Enrollment for Magrolimab Trials; FDA Approval to Incannex’s Sleep Apnoea Clinical Trial; FDA Orphan Drug Designation to Avidity’s AOC 1044; Orphan Drug Designation to CanariaBio’s MAb-AR20.5

Pharma News for Janssen, Galera, Genprex

J&J’s 2-in-1 Tablet for Prostate Cancer; FDA Approves TALVEY for Heavily Pretreated Multiple Myeloma; PDS Biotech Updated on VERSATILE-003 Trial; FDA Issues CRL to NDA for Avasopasem in Radiotherapy-Induced Severe Oral Mucositis in HNC; FDA Orphan Drug Designation to Genprex’s REQORSA; FDA Orphan Drug Designation to Bloomsbury’s BGT-OTCD

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