Rare Disease

Rare diseases–also go by the name Orphan diseases–are the conditions that do not affect many individuals out there in one particular geographic area. Going deeper into the definition, it also means a lesser patient pool for a therapy developed for one identified rare condition. Unfavorable drug uptake would automat...

Lack of knowledge about conditions, situations, or body ailments that are unexplainable often scares people and leads to people jumping to conclusions or say myths that are not at all backed by science and logic. Myths are a seemingly convenient way to explain what the mind perceives as unexplainable.  This...

Like troops at the forefront of any battle, our immune system continually marches to protect us from foreign elements and invaders, including bacteria, viruses, and others. Whenever with a swift wave of wind, something falls in our eyes, our immune system produces tears to get rid of the invader. Or at times when w...

How is a Rare disease defined? Well, it depends on the geography in question, since there is no universal definition, even so, the definition revolves around the point of prevalence or incidence of rare disease. If we talk about the United States, a rare disease affects fewer than 200,000 people (Orphan Drug Act). ...

The Keratoconus market will be dominated by lenses, cross-linking procedures as well as IVMED-80, if approved in the coming decade. Keratoconus, away from the general perception of a rare disease, has come a long way. Thirty-four years back, in Minnesota, United States, this disease was claimed as rare, which cr...

AI-driven treatment approach and growing Fragile X Syndrome market size Healx Pharma has recently partnered with Boehringer Ingelheim to advance the treatment market and invest in R&D related to rare neurological disorders. Under the deal signed, Healx Artificial Intelligence (AI) drug discovery platfo...

A leading global international investment firm, Omega Funds has announced the closure of its sixth fundraising USD 438 Million. More than decade old Life Sciences-based investment firm specialized in direct secondary transactions, Omega funds focuses in investing in Life Sciences vertical, aiming to fulfil the ...

Gaucher Disease Market is anticipated to enhance in the forecast period owing to a strong Gaucher disease pipeline. According to the National Organization of Rare Diseases, Gaucher disease is a rare, inherited, metabolic disorder, which is characterized by an accumulation of harmful fats, chiefly the glycolipid ...

Biohaven buys PRV from GW Pharmaceuticals for USD 105 million GW Pharmaceuticals has announced its decision to sell its rare pediatric disease priority review voucher (PRV) to Biohaven Pharmaceuticals. The voucher was issued under U.S. Food and Drug Administration programme to accelerate speedy recovery of rare ...

Rare diseases are mostly serious, chronic and life-threatening, associated with both psychological as well as financial burdens. In addition, only a few of them have effective drug treatment available. The European Union (EU) definition of a rare disease is one that affects fewer than 5 in 10,000 people. These affe...