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 American Cancer society estimates that in 2019, there will be around 22,530 new diagnoses of Ovarian Cancer and 13,980 women will succumb to the disease. It is the fifth largest cause for death in women, with over 1 in 78 chances of acquiring this...
Earlier this year, we had covered some path-breaking companies engaged in clinical trials, to test the efficacy and safety of novel therapeutics, which could help in the treatment and management of Duchenne Muscular Dystrophy (DMD). Capricor Therapeutics, (NASDAQ: CAPR) a clinical-stage biotechnology company at the forefront...
Mustang Bio’s (NASDAQ: MBIO) shares rose by almost 250% after its development partner, St Jude Children’s Research Hospital in Memphis, published study results that demonstrated that its experimental gene therapy could cure “Bubble Boy” disease or X-SCID, a severe combined immunodeficiency in infants. Figure 1 - Stock...
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...
Parkinson’s is the second most common age-related neurodegenerative disorder, characterized by muscle rigidity, bradykinesia, cognitive impairment, etc. An estimated one million people in the USA are currently battling this disease, with over 60,000 new diagnoses each year. The cost of healthcare is a staggering $25...
