Genetic Disorders

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Notizia - Recent Pharma, Healthcare and Biotech Happenings

Celltrion Announces FDA Nod for New STEQEYMA Presentation to Broaden Pediatric Use; CSL’s ANDEMBRY Gets FDA Nod as First Factor XIIa Inhibitor for Hereditary Angioedema; Ocugen’s OCU410ST Cleared by FDA to Begin Phase II/III Trial in Stargardt Disease; Milestone Pharmaceuticals Submits FDA Response for CARDAMYST in PSVT; AbbVie’s MAVYRET Approved by FDA as First and Only Therapy for Acute Hepatitis C

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Thymidine Kinase 2 Deficiency Treatment: Advances in Therapeutic Strategies and Clinical Development

Types of Mitochondrial Disorders and the Science Behind Them: A Journey from Dysfunction to Discovery

Types of Mitochondrial Diseases and the Science Behind Them: A Journey from Dysfunction to Discovery

pharma-news-for-beam-incannex-kura

Beam’s BEAM-302 Earns FDA Orphan Drug Tag for AATD; Incannex’s IHL-42X Moves to Phase III After FDA Protocol OK; Kura and Kyowa Kirin’s Ziftomenib Gets FDA Priority Review in NPM1-Mutant AML; GSK’s Linerixibat NDA Accepted by FDA for PBC-Linked Pruritus; Ascendis’ TransCon CNP Granted FDA Priority Review for Achondroplasia

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Everything You Need to Know About Niemann-Pick Disease – From Types to Therapies

artificial-intelligence-in-healthcare

Most Promising Artificial Intelligence Applications in the Healthcare Segment

spinal-muscular-atrophy-therapies-in-development

7 Emerging Spinal Muscular Atrophy Therapies Offering Hope for Patients

pharma-news-for-verastem-thermosome-capsida

Eli Lilly’s ZEPBOUND Surpasses WEGOVY in SURMOUNT-5 Trial; Verastem Oncology Secures FDA Approval for AVMAPKI + FAKZYNJA in KRAS-Mutant Ovarian Cancer; Thermosome Gains Orphan Drug Designation for THE001; Beam Therapeutics Receives RMAT Status for BEAM-302 in AATD; Capsida Biotherapeutics Gets IND Clearance for STXBP1 Gene Therapy

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Spinal Muscular Dystrophy Treatment: A Deep Dive into Current Therapies and Future Prospects

evolving-congenital-adrenal-hyperplasia-treatment

Congenital Adrenal Hyperplasia Treatment in Transition: Innovations, Setbacks, and Strategic Shifts Shaping the Market

miplyffa-vs-aqneursa-in-niemann-pick-disease-treatment

New Era in Niemann-Pick Disease: A Comparative Review of MIPLYFFA and AQNEURSA

pharma-news-for-regeneron-gilead-neuronos

FDA Approves Sanofi/Regeneron’s DUPIXENT as First New CSU Therapy in Over a Decade; Gilead’s TRODELVY + KEYTRUDA Shows PFS Benefit in PD-L1+ TNBC; Tempest’s TPST-1495 Gets FDA Orphan Tag for FAP; uniQure’s AMT-130 Granted FDA Breakthrough for Huntington’s; NeuroNOS’ BA-102 Secures FDA Orphan Status for Phelan-McDermid Syndrome

pharma-news-for-cellenkos-mirum-soleno

Cellenkos’ CK0801 Granted FDA Orphan Drug Status for Aplastic Anemia; Mirum’s LIVMARLI Gets FDA Nod for Tablet Formulation; SynOx’s Emactuzumab Earns FDA Fast Track for TGCT; Bristol Myers Squibb’s OPDIVO + YERVOY Combo Approved by FDA for First-Line Advanced Liver Cancer; Soleno Launches VYKAT XR, the First FDA-Approved Therapy for PWS-Related Hyperphagia

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Prader-Willi Syndrome Drug Market Heats Up: 5 Upcoming Challengers to Soleno’s VYKAT XR

pharma-news-for-amgen-novartis-biogen

Amgen’s UPLIZNA Wins First FDA Nod for IgG4-Related Disease; Aldeyra Gets FDA CRL for Reproxalap in Dry Eye; Novartis’ Vanrafia Snags Accelerated FDA OK for IgAN Proteinuria; Biogen’s BIIB080 Earns Fast Track for Alzheimer’s; Epicrispr’s EPI-321 Cleared by FDA for FSHD

evolving-prader-willi-syndrome-treatments

Advances in Prader-Willi Syndrome Treatment: New Hope for Patients

pharma-news-for-beam-astrazeneca-sanofi

Sanofi’s Qfitlia Becomes First FDA-Approved Therapy for Hemophilia A or B; FDA Approves AstraZeneca’s IMFINZI as First Perioperative Immunotherapy for Bladder Cancer; Beam Therapeutics’ BEAM-302 for AATD Receives FDA Clearance; AstraZeneca’s CALQUENCE Combo Greenlit in EU for Mantle Cell Lymphoma; Ionis’ WAINZUA Wins EU Approval for Hereditary Transthyretin Amyloidosis

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