Thanks for the Complement: A New Approach to Treating Rare Diseases


(DGIwire) – The human immune system is a fascinating and complex entity. One of its divisions—called the innate immune system—has a range of powers, including the ability to activate antibodies and tag dubious cells for destruction. According to MedCityNews, this feat is accomplished courtesy of a set of biological processes known as the complement system. […]

Rare Disease Market Revs Up

Young scientist pipetting in life science laboratory.

(DGIwire) – When a disease affects fewer than 200,000 Americans, it is considered “rare.” According to PhRMA, 30 million Americans—10 percent of the population—have one of the 7,000 known rare diseases. Rare diseases have been a focus of drug development for decades, and since the 1980s, there have been financial, legal and legislative incentives—such as […]

Forging a New Direction for Biotech Success

Biotechnology concept

(DGIwire) – “Fail forward” is a mantra among entrepreneurs who are looking for the best way ahead in their business dealings. But it can also describe a strategy adopted by various companies—such as biotechs that have successfully pivoted to a new drug when an old one proved unpromising. Gur Roshwalb, M.D., CEO of Akari Therapeutics, […]

Keeping Track of a Rare Blood Cell Disorder

Pipette mit Tropfen

(DGIwire) – One of the best ways to improve understanding of a disease is to collect comprehensive data from a wide swath of the population of patients who have it. This is certainly true regarding rare diseases for which treatment options are currently limited. One of the more ambitious undertakings of this sort in recent […]

A Battle Against a Blood Disease Enters a New Phase

Close up of two female doctors using tablet and mobile phone

(DGIwire) – It’s one 14-syllable term very few people hear from their doctor: paroxysmal nocturnal hemoglobinuria (PNH). An ultra-rare, life-threatening and debilitating disease of the blood, its symptoms can include intermittently dark urine, fatigue, muscle weakness, abdominal pain, back pain, thrombosis (blood clots), easy bruising or bleeding, headache and shortness of breath, according to the […]

Brown Pee?! Read This

Man looking in the toilet bowl

(DGIwire) – Imagine going to the bathroom and noticing the color of urine is darker than it’s ever been before—almost a tea-colored, dark brown. It might be nothing to worry about—but there is a slight chance it could be a symptom of a serious condition. Known paroxysmal nocturnal hemoglobinuria (PNH), it’s an ultra-rare, life-threatening and […]

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