March 10, 2017 – A new gene that can lead to sudden death among young people and athletes has now been identified by an international team of researchers. The gene, called CDH2, causes arrhythmogenic right ventricle cardiomyopathy (ARVC), which is …

CDH2 gene to cause sudden death in young people Read more »

September 30, 2016 – This is very good news for patients suffering from any form of a rare disease. The European Medicines Agency (EMA) and the American Food and Drug Administration (FDA) have engaged in a joint effort to boost development of …

Rare diseases: EU and US collaborate to boost development of therapies Read more »

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. However, be …

Eteplirsen (Exondys 51) for Duchenne Muscular Dystrophy (DMD) approved Read more »

April 02, 2016 – The European Medicines Agency (EMA) has recommended granting a marketing authorisation in the European Union (EU) for a new gene therapy for the treatment of patients with adenosine-deaminase-deficient severe combined immunodeficiency (ADA-SCID), who have no matching donor for a stem cell …

New hope for children with the ultra-rare immune disorder ADA-SCID: Gene therapy Strimvelis recommended for approval by EMA’s CHMP Read more »

December 12, 2014 – The American Food & Drug Administration (FDA) has recently issued a warning that the antipsychotic drug ziprasidone (marketed under the brand name, Geodon, and its generics) is associated with a rare but serious skin reaction that can …

Rare But Potentially Fatal Skin Reactions (DRESS Syndrome) with Ziprasidone [Geodon] Read more »

April 23, 2014 – The U.S. Food and Drug Administration today approved Sylvant (siltuximab) to treat patients with multicentric Castleman’s disease (MCD), a rare disorder similar to lymphoma (cancer of the lymph nodes). MCD causes an abnormal overgrowth of immune …

FDA approves Siltuximab [Sylvant] for rare Castleman’s disease Read more »

March 19, 1014 – We just learned from a recent Press Release by the American Food and Drug Administration (FDA) on the approval of Metreleptin [Myalept] to treat leptin deficiency. Read the article below: On February 24, 2014, the U.S. …

FDA approves Metreleptin [Myalept] to treat rare metabolic disease Read more »