CRISPR/Cas9 for the Treatment of Duchenne Muscular Dystrophy: Prime time already?

December 12, 2018 – The application of CRISPR/Cas9 based molecular technology in the field of gene editing (or genome editing) has recently had its exploded limelight exposure for a couple of reasons. The question arises if this exposure is earned or somewhat premature.

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First ever treatment for Lambert-Eaton myasthenic syndrome (LEMS) FDA approved

December 04, 2018 –  The American Food and Drug Administration (FDA) has just approved its first ever treatment, Amifampridine (Firdapse), for Lambert-Eaton myasthenic syndrome (LEMS), a rare autoimmune disorder for adult patients. LEMS affects the connection between nerves and muscles and causes weakness and other symptoms in affected patients. 

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Living theragenomic medicine: Direct to consumer test for metabolism of drugs FDA approved

November 08, 2018 –  The American Food and Drug Administration (FDA) has just authorized the first direct-to-consumer test for detecting genetic variants in genes that may govern the metabolism of drugs in individual patients.

Thus, FDA permits marketing, with special controls,

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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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Cancer therapies: Immune checkpoint inhibitors may kill you

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Diabetes: No end to SGLT2-inhibitor drugs side effects

01 September 2018 – The American Food and Drug Administration (FDA)  is warning that cases of a very rare but serious infection of the genitals and area around the genitals have been reported with the class of type 2 diabetes medicines called sodium-glucose cotransporter-2 (SGLT2) inhibitors.

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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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Risk of Bullous Pemphigoid with Type 2 Diabetes Drugs

August 12, 2018 – Findings from a retrospective case-control study were published online August 8, 2018, in JAMA Dermatology indicate that the use of certain dipeptidyl peptidase 4 (DPP-4) inhibitor drugs are associated with a small but significantly elevated risk for developing bullous pemphigoid,

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Ivosidenib (Tibsovo) for AML-patients with IDH1-mutations

July 22, 2018 – The American Food and Drug Administration (FDA) just approved Ivosidenib (Tibsovo) for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) who have a specific genetic mutation in the IDH1 gene.  

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Coffee Drinking, Genetic Variation, and Mortality

July 07, 2018 – A very recent paper by Loftfield E. et al., published online in JAMA Intern. Med. on July 02, looked at the association of coffee drinking with mortality by genetic variation in caffeine metabolism.

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