Pharm-Olam Blog

March was Rare Disease Month at Pharm-Olam. We started off by reviewing an article that was published in Nature (read it here). From there, we examined the field of rare disease research (click here). We went on to look at the specific drug classes and development platforms that researchers use in rare disease research. We covered small molecules (access it here), antibody therapy (available here), protein replacement therapies (read it here), oligonucleotide therapies (read it here), gene therapies (click here to read it), and drug repurposing (available here).

Rare Disease Day may have been on 29 February, but at Pharm-Olam, we think it desires an entire month. Since the beginning of March, we have been focusing on rare disease research. We reviewed an article from Nature (click here to read it) and took a closer look at rare disease research as a whole (read it here). From there, we reviewed small molecules (click here), antibody therapy (access it here), protein replacement therapies (available here), oligonucleotide therapies (click here for the article), and gene therapies (read it here).

At Pharm-Olam, March is Rare Disease Month. Over the past three weeks, we have been exploring the subject in greater detail. We started with an article that had appeared in Nature (read it here) and then started looking at rare disease research (click here). So far, we have covered small molecules (access it here), antibody therapy (read it here), protein replacement therapies (click here for the article), and oligonucleotide therapies (available here).

Rare disease research is such a complicated area because it involves every therapeutic area individually and sometimes in unique combinations. Being able to develop treatments for these conditions requires a strong knowledge of those areas of the body that are impacted and the way that different drug platforms can influence those functions. 

Rare disease research can take many forms, and each drug development platform offers specific advantages researchers can leverage to address elements of these conditions. Earlier this month, we began to discuss research within the field of rare disease (read the article here). We reviewed an article from Nature (you can access it here) and went in detail about specific types of rare disease research in subsequent articles. At this point, we have covered small molecules (click here) and antibody therapy (read it here). In this article, we are going to take a closer look at protein replacement therapies in rare disease research.

Today, we continue our look into rare disease research. At Pharm-Olam, March is rare disease month. Last week, we began our examination of the subject (read the article here). In that article, we looked at rare disease in detail and explained how common rare diseases actually are with over 7,000 known diseases. We also explored some of the common origins for known rare diseases and explained how this knowledge helps researchers develop treatments for those conditions using small molecules (click here). We looked at the small molecule platform and its opportunities in the fight against rare disease.  In this article, we will look at the use of antibody therapy in rare disease research.

Earlier this week, we looked at rare disease research in the broadest terms (read the article here). We explained that rare disease is a classification that is comprised of thousands of different conditions, but, collectively, the number of people with rare diseases is quite significant. Most of these conditions are caused by inheriting the right combination of genes to bring a recessive condition to the surface, but there are other possibilities, including illness and allergies. Understanding this root cause enables researchers to develop treatments.

Clinical research has grown in leaps and bounds. Researchers now have the tools to understand many of the world’s rarest diseases at a molecular level. At the same time, current legislation supports the development of treatments to address these rare conditions. However, challenges in this realm persist. A recent article in Nature reviewed the challenges in rare disease development as well as the opportunities that different rare disease development platforms present.