Rare Diseases Get Their Turn in the Spotlight

Caucasian female nurse wearing a protective mask, close up

(DGIwire) – Held on the last day of February each year, Rare Disease Day shines a spotlight on an issue that affects as many as 30 million Americans, according to the National Institutes of Health. There are about 7,000 rare diseases, reports PharmaVOICE, most of which are genetic in origin; in the U.S., a disease […]

4 Insects Used to Treat Wounds and Diseases

Tick in Spiderweb

(DGIwire) – Think bugs are annoying and have no redeeming qualities? Well, think again. A slew of research has exposed an amazing fact: insects can help address a wide variety of human ailments. More are being studied for use to treat wounds and diseases. Here are four examples of how insects have been shown to alleviate […]

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

Tick

(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 […]

Dry Eyes? Dry Mouth? It Could Be Sjögren’s Syndrome

dry-eyes

(DGIwire) – Everyone experiences dry eyes and mouth from time to time, but when these symptoms persist—especially in tandem with pain and fatigue—they might be symptoms of a more serious underlying condition: Sjögren’s syndrome. More than four million Americans suffer from this autoimmune disorder, according to the Sjögren’s Syndrome Foundation (SSF), so it pays to […]

A Push to Boost Funding for Rare Diseases

Focused on finances

(DGIwire) – There are 7,000 rare diseases that impact the lives of 30 million Americans, but 95 percent of them still have no treatment, according to the National Organization for Rare Disorders (NORD). Now—inspired by a $2 billion boost in funding given to the National Institutes of Health (NIH) by Congress for fiscal year 2016—NORD […]

Newly Diagnosed with PNH? 4 Things to Do Now

Doctor and patient shaking hands

(DGIwire) – Those who have been newly diagnosed with paroxysmal nocturnal hemoglobinuria (PNH) may be going through a confusing and emotional period. However, there are several steps they can take to deal with their diagnosis. Here are a few suggestions: Learn more about PNH: PNH is a rare and serious acquired disease that causes red […]

Keeping Track of a Rare Blood Cell Disorder

Hand with latex glove holding blood sample on white background

(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 […]

Orphan Drugs: What Are They?

blood sample

(DGIwire) – Anyone who spends time browsing through healthcare news stories is likely to come across the term “orphan drug.” But what is an orphan drug? According to the U.S. Food and Drug Administration (FDA), it grants Orphan Drug Designation status to products that treat rare diseases, providing incentives to sponsors developing drugs or biologics. […]

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 […]

Injecting a New Strategy to Treat a Rare Blood Disease

Male Patient Having Consultation With Doctor In Office

(DGIwire) —  Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-rare, life-threatening and debilitating disease of the blood. According to the National Institutes of Health, the symptoms of PNH can include intermittently dark urine, abdominal pain, back pain, thrombosis (blood clots), easy bruising or bleeding, headache and shortness of breath. The treatment of PNH is directed at […]

Why Is the Zika Virus Leaving Some Patients Paralyzed?

Mosquito on white background

(DGIwire) – Although concerns that the Zika virus will cause serious birth defects have grabbed most of the headlines, The Lancet recently reported evidence that an encounter with the virus can leave some people temporarily paralyzed. In March 2016, the journal published the first systematic study of a possible link between the virus and . […]

A Teary-Eyed Look for Clues About an Autoimmune Disease

Young woman with problems

(DGIwire) – Tears of sorrow could turn to tears of joy if scientists’ work on an autoimmune disease bears fruit. In June 2016, Ophthalmology Times reported that research on the composition of tears is generating insights that could lead to earlier detection and treatment of Sjögren’s syndrome— an inflammatory disease that can affect many parts […]

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