Blog Archives

Hope for patients with cutaneous squamous cell carcinoma (CSCC): Cemiplimab-Rwlc (Libtayo)

October 31, 2018 – The American Food and Drug Administration (FDA)  recently approved Cemiplimab-Rwlc (Libtayo) injection for intravenous use for the treatment of patients with metastatic cutaneous squamous cell carcinoma (CSCC) or locally advanced CSCC who are not candidates for curative surgery or curative radiation.

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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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  • Distinct social profile and high ASD risk, 3q29 deletion survey finds July 19, 2019
    A survey of 93 people with 3q29 deletion syndrome reveals a distinct pattern of social disability and anxiety, even without a diagnosis of autism spectrum disorder. The results were published online in Molecular Autism on July 16.
  • Study sheds light on the darker parts of our genetic heritage July 19, 2019
    More than half of our genome consists of transposons,DNA sequences that are reminiscent of ancient, extinct viruses.Transposons are normally silenced by a process known as DNA methylation, but their activation can lead to serious diseases. Very little is known about transposons but researchers in an international collaboration project have now succeeded for the first time […]
  • Molecular cause for severe multi-organ syndrome July 19, 2019
    Three unrelated families on three continents (from continental Portugal, the United States and Brazil), all with healthy ancestors, had children with a very rare multi-organ condition that causes early-onset retinal degeneration, sensorineural hearing loss, microcephaly, intellectual disability, and skeletal dysplasia with scoliosis and short stature.
  • Researchers determine epigenetic origin of docetaxel-resistant breast cancer July 19, 2019
    Researchers at the Bellvitge Biomedical Research Institute (IDIBELL) have published a study in Molecular Cancer Research in which they identify methylation patterns associated with different subtypes of breast cancer, and a subclassification of the group of "triple negatives," a breast cancer type typically associated with poor prognosis. In addition, they identified changes in DNA methylation […]
  • Researchers report the function of reverse-twisting DNA July 19, 2019
    Normally, the two strands of the DNA double helix wind around each other in a right-handed spiral. However, there is another conformation called Z-DNA in which the strands twist to the left. The function of Z-DNA has remained a mystery since its discovery. A newly published paper unambiguously establishes that the Z-conformation is key to […]
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