Rare Disease

Oct 16, 2024

Pompe disease is a rare genetic disorder that presents a spectrum of severity, with varying rates of progression and ages of onset. Symptoms can appear anywhere from infancy to late adulthood, with earlier onset generally associated with more rapid progression and increased severity. At all ages, the disease is mar...

Jan 05, 2024

Ionis Pharmaceuticals and AstraZeneca concluded the year 2023 on a positive note as they received the eagerly awaited approval from the FDA for their transthyretin amyloidosis (ATTR) medication, Wainua. In particular, the agency approved the use of the ligand-conjugated antisense oligonucleotide (LICA) medication f...

Oct 16, 2023

Rare diseases affect a larger population than cancer and AIDS combined, with over 7,000 distinct conditions impacting more than 300 million globally. Approximately 1 in 10 individuals are affected by a rare disease, and it often requires several years to receive an accurate diagnosis, averaging around 4.8 years. Ap...

Jun 16, 2023

Dystrophic epidermolysis bullosa (DEB) is an inherited disorder caused by changes in the COL7A1 gene. In this rare condition, blisters form on the skin and the moist inner lining of some organs and body cavities. Depending upon the nature of the inheritance pattern, dystrophic epidermolysis bullosa is divided into ...

May 08, 2023

In recent years, the development of cell and gene therapies has provided a new avenue for the treatment of rare disorders. This article will discuss the current and future scenarios of cell and gene therapies in rare disorders with cancer indications as an exception, in Japan. Cell therapy is a treatment in which l...

Apr 20, 2022

Drug pricing is one of the hottest topics in the healthcare segment. Several arguments have been put forward by people, governments, healthcare companies, and other organizations in favor of and against expensive medicines. Some of the drugs are so expensive that they are nearly out of reach of the common people. H...

Mar 21, 2022

The universality of mobile technology has proven to be extremely beneficial, specifically in recent years. The potential of improving medical care delivery, and the potential for instantaneous and two-way transfer of information has turned out to be a revolutionary step. The following article indicates the importan...

Feb 09, 2022

Artificial Intelligence (AI), with its wide range of applications, is enhancing the speed and accuracy of businesses across various industries such as manufacturing, healthcare, finance, marketing, and several others. To take advantage, globally, companies, whether big or small, are actively adopting artificial int...

Dec 07, 2021

Aslan Pharmaceuticals enters into a strategic collaboration with IQVIA   ASLAN Pharmaceuticals announced the appointment of IQVIA Biotech, a provider of analytics, technology solutions, and clinical research services to the life sciences industry, as its preferred clinical research organisation for log...

May 25, 2021

TLX66 Meets Study Objectives, Holding Out in AL Amyloidosis Telix Pharmaceuticals reported the results from the TRALA trial (Targeted Radiotherapy for AL Amyloidosis), a Phase I/IIa trial evaluating the safety and toxicity of TLX66 as the sole bone marrow conditioning agent prior to autologous hematopoietic stem...