Genetic Disorders

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

Notizia - Recent Pharma, Healthcare and Biotech Happenings

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

FDA Approves BeiGene’s Brukinsa for CLL/SLL BeiGene's Brukinsa (zanubrutinib) for chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) has been approved by the US Food and Drug Administration. CLL is a common type of leukemia, accounting for approximately 25% of all new cases each year. SLL is a much rarer form of cancer than CLL, but the malignant cells are concentrated in the lymph nodes rather than the blood and bone marrow. In the SEQUOIA trial, 479 patients were randomly assigned to either zanubrutinib until disease progression or unacceptable toxicity or bendamustine plus rituximab (BR) for six cycles. An independent review committee (IR...

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Charcot-Marie-Tooth Disease Market Size Expects Growth Owing to Increasing Prevalence, Influx of Pharma Companies and Novel Therapies

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Shape Therapeutics-Roche’s Deal; AllStripes Raises $50M; Datavant-Real Chemistry’s Partnership; BlueWillow’s Nasal Vaccine

Von Hippel-Lindau Market Space Expected to Expand at a CAGR 8.3% by 2030 Owing to Increasing Prevalence and Emerging Therapies

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Asher Bio raises $55M; Roche halts Huntington’s phase 3 trial; Novartis’ radioligand hits goal in phase 3; EpimAb raises $120M in Series C

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Genetic Disorders

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

Charcot-Marie-Tooth Disease Market Size Expects Growth Owing to Increasing Prevalence, Influx of Pharma Companies and Novel Therapies

Shape Therapeutics-Roche’s Deal; AllStripes Raises $50M; Datavant-Real Chemistry’s Partnership; BlueWillow’s Nasal Vaccine

Von Hippel-Lindau Market Space Expected to Expand at a CAGR 8.3% by 2030 Owing to Increasing Prevalence and Emerging Therapies

Primary Hyperoxaluria Market Size Observes Growth with a Substantial CAGR

Angelman syndrome Pipeline: Unmet needs call for a robust pipeline

TLX66 in AL Amyloidosis; Takeda’s Dengue Shot; Innovent’s Cholangiocarcinoma Clinical Trial; Another Failed NASH Drug

Who is Going to be a Trendsetter in the Hereditary Deafness Market?

Abingworth & Alebund’s Finacial Closing; Pfizer/BioNTech COVID-19 Vaccine Expanded Use; Biogen and Capsigen Deal

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Rising Awareness, Key Companies, Emerging Therapies? What is Shaping the Alagille Syndrome Market

Alpha-1 Antitrypsin Deficiency: Symptoms, Treatments and Key Companies in the Market

Asher Bio raises $55M; Roche halts Huntington’s phase 3 trial; Novartis’ radioligand hits goal in phase 3; EpimAb raises $120M in Series C

Hemophilia B Market: How Pipeline Therapies are Transforming the Treatment Hemisphere?

A Royal Disease: Hemophilia

Emerging Research Continues To Transform Erythropoietic Protoporphyria Market Outlook

Biohaven’s Troriluzole Failure; Daiichi/ AZ’s Enhertu; Fujifilm & Manufacturing Spree; J&J’s Darzalex Faspro

Angelman Syndrome Market: Insights Into The Recent Late-Stage Drug Failures And Novel Approaches To Treating This Rare Neurogenetic Disorder

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