Blog Archives

Patisiran (Onpattro): First targeted RNA-based therapy approved to treat hATTR

August 14, 2018 – The American Food and Drug Administration (FDA) just approved Patisiran (Onpattro) infusion for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients.

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Cancer is not like cancer: Ethnic background matters

November 24, 2017 – New research indicates that one-size treatment for one form of blood cancer likely does not fit all, particularly when it comes to ethnic differences of patients.  Thus, African-American men are three times more likely to be diagnosed with multiple myeloma,

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Besponsa: Is this really a viable new treatment for ALL?

August 18, 2017 – B-cell precursor acute lymphoblastic leukemia (ALL) is a aggressive and rapidly progressing type of cancer in which the bone marrow makes too many B-cell lymphocytes, an immature type of white blood cell. In the United States, the National Cancer Institute estimates that approximately 5,970 people will be diagnosed with B-cell ALL this year and approximately 1,440 will die from the disease.

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Eteplirsen (Exondys 51) for Duchenne Muscular Dystrophy (DMD) approved

September 19, 2016 – This post presents below a virtually unedited announcement by the American Food & Drug Administration (FDA) concerning Duchenne Muscular Dystrophy (DMD) therapy  option which gives much hope for at least some of the affected patients.

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The HCV saga goes on: Epclusa has been approved for treatment of chronic Hepatitis C virus (HCV) infection

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Hope for patients with urothelial carcinoma: Atezolizumab (Tecentriq) approved

May 18, 2016 –  Over the last two years, biologics  targeting the PD-1/PD-L1 pathway (proteins found on the body’s immune cells and some cancer cells), notably Nivolumab (Opdivo) and Pembrolizumab (Keytruda), have come to the clinic for the treatment of a selection of oncologic indications.

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Venetoclax (Venclexta) for the treatment of patients with chronic lymphocytic leukemia (CLL)

April 12, 2016 – The American Food and Drug Administration (FDA) just approved Venetoclax (Venclexta) for the treatment of patients with chronic lymphocytic leukemia (CLL) who have a chromosomal abnormality called 17p deletion and who have been treated with a least one prior therapy.

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Theragenomic Medicine: Gefinitib (Iressa) targeted therapy for first-line treatment of patients with a type of metastatic lung cancer approved

July 13, 2015 – This post persents a press announcement by the U.S. Food and Drug Administration (FDA).  The text has slightly been edited by the autor of this blog. Thus, FDA today approved Gefinitib (Iressa) for the first-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors harbor specific types of epidermal growth factor receptor (EGFR) gene mutations,

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Theragenomic Medicine: Targeted Therapies and Associated Toxicity

March 22, 2015 – This post addresses a topic which often gets lost in the current hype on targeted and precision (theragenomic) medicine. The issue is that targeted therapies are not only very precise and highly effective (in most patients) but they often also come with considerable clinical toxicity.

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Theragenomic Medicine: Palbociclib (Ibrance) approved for postmenopausal women with advanced (metastatic) breast cancer

February 8, 2015 – The American Food and Drug Administration (FDA) has granted accelerated approval to Palbociclib (Ibrance) to treat advanced (metastatic) breast cancer.  The FDA granted Ibrance breakthrough therapy designation because the sponsor demonstrated through preliminary clinical evidence that the drug may offer a substantial improvement over available therapies.

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