AzurRx BioPharma (NASDAQ: AZRX), a development stage company that is developing oral, nonsystemic, recombinant proteins for the treatment of gastrointestinal diseases, announced the results from a Phase II Clinical Trial of MS1819. The phase II trial named OPTION was an open-label, crossover trial being carried...
CRISPR, which is an acronym for Clustered Regularly Interspaced Short Palindromic Repeats, is a revolutionary genome editing approach that helps change an individual’s DNA, by altering it at a specific location. The Crisper-Cas9, short for clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9,...
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...
Niemann–Pick is a rare autosomal recessive inherited disease that is characterized by excessive accumulation of cholesterol and lipids in cells, leading to apoptosis and neural degeneration. The disease affects numerous body parts such as the brain, nerves, liver, spleen, bone marrow and lungs. Those suffering...
Sjogren’s, named after its discoverer Henrik Sjögren, is the most prevalent systemic autoimmune disease that predominantly affects women. This disorder is manifested in the form of dry eyes, dry mouth, joint pains and chronic fatigue and if left untreated can cause severe dysfunction in organs...
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...
