Medscape recently published an article stating that the EMA is conducting a review of rucaparib, a drug used to treat patients with a range of ovarian cancers that have relapsed. These…
Chinese patients living with pediatric growth hormone deficiency are one step closer to a new treatment option, according to a recent article from PharmaBiz.com. The step I'm referring to is…
Acromegaly is a rare disease that is characterized by excess and abnormal levels of growth hormone (GH). Because of this, medical professionals are very interested in these levels as patients…
Data based on approximately five million people suggests that esophageal cancer has nearly doubled from 2012 through 2019. According to a recent report in MSN, the incidence of precancerous Barrett’s…
It’s no secret that living with a rare or underserved condition can come alongside a heavy financial burden. A recent study supported by the National Institutes of Health (NIH), Cure…
In April 2022, Dr. Adrián Alambiaga successfully defended his doctoral thesis at CEU Cardenal Herrera University. His thesis centered around the exploration of progesterone as a way to stop cell…
In a news release from late May 2022, pharmaceutical company SteroTherapeutics LLC, known for developing orphan drugs for rare conditions, shared that its novel therapy ST-003 received Orphan Drug designation…
Rare disease research can be difficult to conduct, with one of the major contributing factors being funding. Research is expensive, and I mean really expensive. That's why grant funding and other…
Gannex has just announced that they have dosed the first patient in their Phase 2 clinical trial examining ASC42 as a treatment for those diagnosed with primary biliary cholangitis (PBC).…
When they found out that they were having a son, Nash and Callie Horne were thrilled. They even decided to name their son Nash as well. But then an unexpected…
Fast Track designation is designed to facilitate the development of, and expedite the review of, drugs intended to treat serious conditions and fill unmet patient needs. This designation comes alongside…
Drug development is a long, expensive, and difficult process. Not every treatment being created makes it to the end of the clinical trial process, much less through FDA approval. Takeda…
In the past, researchers have observed a relationship between hemophilia and bone-related issues. However, while this observation is helpful, there have been few studies examining the prevalence of bone problems…
The WHO’s Emergency Response System Has Been Activated. Just last month, a World Health Organization (WHO) representative assured VICE World News that the WHO is taking this global outbreak very…
When a patient or caregiver speaks of immunotherapy it is often with a more confident tone than when they are speaking about chemotherapy. However, the odds of a patient going…
ITP, or immune thrombocytopenia, is a platelet disorder where the immune system destroys its own platelets by way of destructive antibodies. Platelets are very small blood cells that originate in…
Imagine the feeling of working for years with the goal of saving children who have congenital athymia. Then thirty years later the FDA approves a regenerative medicine based on your…
Alzheimer’s disease is the primary cause of the progressive decline of cognitive function in the elderly. It is characterized by underlying complications in the central nervous system (CNS). To date,…
LPCN 1144 for nonalcoholic steatohepatitis (NASH) has already demonstrated positive results in the LiFT trial, and it is now showing more exciting results in its open-label extension (OLE) trial. Want…
For over thirty years, researchers at the University of Pittsburgh (UPMC) have been observing cases of limited scleroderma, a connective tissue disorder that causes thick hard patches of skin. Scleroderma…
How does our body fight infections? After we get infected with a serious illness, in what ways does our immune system prepare? Understanding these processes could help hone treatments and…
Adolescence is the phase which exists between childhood and adulthood; therefore, people who are between ages 10-19 are typically considered to be adolescents. How does adolescence change the way people…
A new study has examined the long-term impacts of esophageal dilation in eosinophilic esophagitis (EoE). The aim was to uncover the link between dilation and symptom reports. Additionally, the research…
Aeterna Zentaris’s ongoing DETECT trial is evaluating the drug macimorelin (tradename Macrilen™) for childhood-onset growth hormone deficiency (CGHD). As recently announced in an article published by GlobeNewsWire, the study (AEZS-130-PO2),…
The Food and Drug Administration (FDA) has four distinct approaches to expedite the availability of therapies for rare, severe, or life-threatening illnesses: Priority Review, Breakthrough Therapy, Fast Track, and Accelerated…
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