Fabry Disease also known as angiokeratoma corporis diffusum or alpha-galactosidase A deficiency, is an inherited disorder characterized by the buildup of globotriaosylceramide, a type of fat in the body’s cells and results in numerous disorders such as acroparesthesias (burning, tingling, or prickling sensation in extremities),...
Sarepta Therapeutics (NASDAQ: SRPT) faced a setback recently with the FDA rejecting Vyondys 53 (Golodirsen), an experimental drug for Duchenne muscular dystrophy, which targets over 8% of the DMD patients with a rare genetic mutation. The company was issued a Complete Response Letter (CRL), which...
The Cannabis plant has an estimated 113 different compounds, out of which THC and CBD are the two main constituents. These affect the receptors in the brain to alter an individual’s physical and mental state. THC is mainly responsible for the psychoactive effects or the...
Systemic lupus erythematosus (SLE) is an autoimmune disease that causes the body’s immune system to attack healthy tissue causing inflammation, photosensitivity, oral ulcers, pleuritis, joint pain, arthritis, fever and a butterfly rash. In the long-run it can affect the kidneys, brain and heart as well....
The Muscular Dystrophy Association describes Duchenne Muscular Dystrophy (DMD) as a fatal, X-linked genetic neuromuscular disorder that leads to progressive, irreversible loss of muscle function, including the heart and lungs. It has an X-linked recessive inheritance pattern and is acquired by a “Carrier” mother. Globally, 1 in...
