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5 Companies unraveling the potential of Gene Based Therapies for the treatment of Sanfilippo syndrome (Mucopolysaccharidosis type III (MPS III)

Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is caused by the mutations in the glucosamine (N-acetyl)-6-sulfatase (GNS), heparan-alpha-glucosaminide N-acetyltransferase (HGSNAT), N-acetyl-alpha-glucosaminidase (NAGLU) and N-sulfoglucosamine sulfohydrolase (SGSH) genes, which are responsible for the synthesis of large sugar molecules called glycosaminoglycans (GAGs). The dysfunction...

04 September, 2019 0 Comments

Kala Pharmaceuticals – CRL for KPI-121 0.25% poses a Setback for the Company

Kala Pharmaceuticals (NASDAQ: KALA) received a Complete Response Letter (CRL) from the FDA, asking for efficacy data from an additional trial, in response to its NDA for KPI-121 0.25%, its lead candidate for the treatment of dry eye disease. The company had filed a New...

03 September, 2019 0 Comments

Seelos Therapeutics – FDA Acceptance of IND for SLS-005 for Sanfilippo Syndrome a shot in the arm for the Company

Seelos Therapeutics (NASDAQ: SEEL), a clinical-stage biotechnology Company, announced the acceptance of its Investigation New Drug (IND) application for SLS-005 (trehalose) by the FDA. The company is planning to initiate a combined Phase IIb/III, multicenter study to assess the safety, tolerability and efficacy of trehalose...

31 August, 2019 0 Comments

Sarepta Therapeutics – Rejection of marketing application for DMD drug leaves the Company flummoxed

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...

29 August, 2019 0 Comments

5 Most Promising Companies offering hope to patients with Niemann-Pick Type C

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...

28 August, 2019 0 Comments

PAVmed Inc – Grant of Patent for Company’s CarpX – A minimally invasive device for treatment of Carpel Tunnel Syndrome

PAVmed, a multiproduct medical device company that is developing solutions for areas with large unmet medical needs, today announced the grant of a patent for its CarpX minimally invasive device that aims at treating carpal tunnel syndrome. The U.S patent 10,335,189 entitled “Systems and Methods...

27 August, 2019 0 Comments

5 Most Promising Companies Riding the Waves of Change in the CDB Biotechnology Space

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...

20 August, 2019 0 Comments

Top 5 Companies in the Hemp-based CBD Market

Hemp and Marijuana, though used interchangeably, are distinct strains of the Cannabis sativa plant and have different phytochemical uses and composition. Hemp has very low concentrations of THC and is rich in CBD, which results in very negligible psychoactive effects. Hemp is extensively used in industrial...

19 August, 2019 0 Comments

Top Innovations in the Treatment of Methylmalonic Acidemia (MMA)

Methylmalonic Acidemia, a rare inherited disease that occurs in an estimated 1 in 50,000 to 100,000 people, is characterized by the inability of the body to process proteins and lipids effectively. This disease mostly affects infants leading to hypotonia, hepatomegaly and in severe cases failure...

10 August, 2019 0 Comments

5 Companies with Potential to Transform the Rosacea Market

Rosacea affects over 415 million people worldwide, and more than 16 million in the U.S alone, according to research conducted by the University of Copenhagen, Denmark. It is a fairly common skin disorder, but only 18 percent of Americans afflicted with this condition are under...

07 August, 2019 0 Comments

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