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Rare Diseases

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A Glance at Key Insights From 42nd J.P. Morgan Annual Healthcare Conclave

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anca-associated-vasculitis-treatment

ANCA-associated Vasculitis Treatment Market: Unraveling the Complexities

gene-therapy-in-rare-disorders-challenges-in-europe

Gene Therapy in Rare Disorders: Acceptance in Europe Faces Challenges

cell-and-gene-therapies-for-rare-disorders-treatment

Watershed Moment for Cell Therapies and Complicated Journey of Gene Therapies in Japan

daybue-for-rett-syndrome-treatment

DAYBUE: First and Only Approved Drug for Rett Syndrome Treatment

Pharma News and Updates for Novartis, Pharming, Cyclerion, Iovance, BPGbio

Novartis Announces the Positive Results of Phase III NATALEE Trial Evaluating Kisqali; FDA Approves Pharming’s Joenja for APDS; FDA Orphan Drug Designation to Cyclerion’s Zagociguat; Iovance Completes BLA Submission for Lifileucel in Advanced Melanoma; BPGbio Announces Partnership with debra of America

Pharma news and updates for Roche, Acadia, Shorla Oncology, Calliditas, Mesoblast, Sanofi, Provention Bio

Sanofi to Acquire Provention Bio; USFDA Committee Votes in Favor Roche’s Polivy; FDA to Review Mesoblast’s GvHD Drug; FDA Approves Acadia’s Daybue ; FDA Approves Nelarabine Injection; Calliditas’s Nefecon Trial Reuslt in IgA Nephropathy

Next-Generation Sequencing Market Trends, Development, Key Companies, Applications, and Future

Evaluating the Key Trends and Developments in the Global Next-Generation Sequencing Market

Pharma News and Updates for SpringWorks Therapeutics, AskBio, Mitsubishi Tanabe Pharma America, Sage Therapeutics, Biohaven Pharma

SpringWorks’s Desmoid Tumors Therapeutic, Nirogacestat; Orphan Drug Designation to AskBio’s AB-1003; Mitsubishi Tanabe’s Phase 3 Trial of RADICAVA ORS in ALS; EU Orphan Drug Designation to Sage Therapeutics’s SAGE-718; FDA Fast Track Status to Biohaven’s Taldefgrobep Alfa

Pharma News and Updates for Actinium, Apellis, Chiesi, IVERIC, Travere, Takeda

Actinium Announces Phase III SIERRA Trial Results; FDA Approves Apellis’s Geographic Atrophy Drug; FDA Gives Green Light to Chiesi’s Lamzede; FDA Accepts NDA and Grants Priority Review for Avacincaptad Pegol; FDA Approves Travere’s Kidney Disorder Drug; Takeda Presents Phase 3 GRAPHITE Study Results

latest-pharma-news-and-updates-for-eli-lilly-novartis-janssen-ipsen-kite

Eli Lilly’s Jaypirca Approval; Novartis’ Adakveo EMA Review; Janssen’s CARTITUDE-4 Study of CARVYKTI; Negative Review on Ipsen’s Palovarotene; Gilead Sciences and Kite’s Yescarta NICE Recommendations; Daiichi Sankyo and AstraZeneca’s Enhertu EU Approval

Latest Pharma News and Updates for BeiGene, Seagen, Alnylam, Roche, Astellas

BeiGene’s Brukinsa Approval; FDA Approval to Seagen’s TUKYSA; NICE Recommends Alnylam’s Amvuttra; FDA Approves Brenzavvy for Type 2 Diabetes; Roche’s Tecentriq to be Filed for Early-stage Liver Cancer; FDA Lifts Hold on Astellas’ Pompe Gene Therapy

behcets-syndrome-treatment-market

Off-label Therapies Dominating the Behcet’s Syndrome Treatment Market

Pharma News and Updates for Gilead, Biogen, Pfizer, MediWound, UCB

Gilead Buys Out Rights to Cancer Therapy from Jounce; FDA Places Clinical Hold on Biogen’s Orelabrutinib; Pfizer Announces Phase 3 BENEGENE-2 Study Result; FDA Approves MediWound’s NexoBrid; UCB Announces FDA Acceptance of BLA Resubmission for Bimekizumab; FDA Approves TG Therapeutics’ Briumvi

myotonic-dystrophy-treatment-market-assessment

Myotonic Dystrophy Treatment: In Search of Effective Weaponry to Kick Out Toxic RNAs Weakening Muscles

Pharma News for Nkarta, Eisai, EQRx, Rigel Pharma, AUM, X4 Pharma

Nkarta’s Anti-CD19 Allogeneic CAR-NK Cell Therapy, NKX019; Eisai Presents Results of lecanemab for Alzheimer’s Disease; EQRx’s Aumolertinib for EGFR-Mutated NSCLC; FDA Approves Olutasidenib for IDH1-Mutated R/R AML; FDA Orphan Drug Designation to AUM302 for Neuroblastoma; X4 Pharma Announces Results for WHIM Syndrome Drug

gene-therapy-for-complex-diseases

Gene Therapy: The Next Milestone in Treating Complex Diseases

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