According to a story from Globe Newswire, the rare disease company Albireo Pharma, Inc. has recently announced the release of new data from its phase 3 trials. These trials were…
According to a story from AJMC, there are a number of factors in play when determining the path forward for the treatment of advanced melanoma. For this story, Dr. Omid…
In the United States, during the drug development and approval process, drug developers are able to request Fast Track designation. This process is designed to facilitate and expedite the…
At the end of June 2022, biopharmaceutical company InflaRx N.V. (“InflaRx”) shared via press release that its product vilobelimab recently earned Orphan Drug designation in both the United States…
A study of hereditary angioedema (HAE) patients in Brazil was featured in an article in the Angioedema News. Researchers found that patients waited on average seventeen years for a…
Individuals with Duchenne muscular dystrophy have mutations in the gene that makes the protein dystrophin. This gene is the largest gene in the human body, with 79 exons. When there…
According to a story from Globe Newswire, olipudase alfa (marketed as Xenpozyme) has been approved by the European Commission for the treatment of non-CNS related symptoms of acid sphingomyelinase deficiency…
In 2019, Courtney and Michael Henderson welcomed the newest member of their family – their son Luke. They were thrilled at this new addition, who they say is one…
According to a recent article, the PI3K inhibitor umbralisib used to treat marginal zone lymphoma has had its approval withdrawn by the FDA. Marginal Zone Lymphoma (MZL) Marginal…
In many cases, orally administered PDE5 inhibitors – such as Cialis, Levitra, and Stendra – have been used to treat erectile dysfunction. These vasodilating drugs were found to be…
The number of Alzheimer’s disease diagnoses has been rising rapidly over the last few decades. Today, an estimated 6.5 million Americans alone are living with this disease. Yet there…
The study was intended to recruit 6,000 Alzheimer's patients, but even after recruiting had proceeded for seven months, just 29 patients were enrolled. As a result, the trial was cancelled,…
According to a recent article, $100 million has been given to four research teams by the Cancer Grand Challenges program for cancer research. Cancer Grand Challenges Program The Cancer…
According to a story from Globe Newswire, the global healthcare company Novo Nordisk recently announced encouraging findings from its phase 3 clinical trial. This study was evaluating somapacitan (marketed as…
Unfortunately, it can sometimes be difficult to get companies to perform research and develop therapeutics for rare diseases, but the FDA came up with a process to spur research…
The FDA has just announced that they have lifted the temporary hold placed on pheNIX gene therapy. This gene therapy was created by Homology Medicine as an investigational treatment for…
According to a story from news-medical.net, the International Rett Syndrome Foundation (IRSF) has announced the launch of its Clinical Trial Committee. The committee was implemented with the goal of helping…
When Brianna Terry was just two years old, her doctors gave her a diagnosis of autosomal recessive osteopetrosis. At the time, the doctors were concerned about her wellness; they…
According to a story from the BHD Foundation, Patient Worthy partner The Myrovlytis Trust, along with the BHD Foundation and Pulse Infoframe, are collaborating on an international Birt-Hogg-Dubé (BHD) syndrome…
According to a story from Scleroderma News, June is recognized as Scleroderma Awareness Month, a time for spreading awareness about scleroderma among the general public and in the medical field.…
Written by: Tyler Bradley, PFIC Network The PFIC Scientific Conference Held Many Great Presentations and Roundtables That Informed Both The Scientific And Patient Communities. Last month the PFIC Network hosted…
According to a recent article, scientists were able to create cilia-free stem cells that may help in finding the causes of cilia-linked illnesses such as polycystic kidney disease. …
Eric Miller was diagnosed with Lyme carditis, which is a very rare complication that can arise from Lyme disease. He is a 43-year-old who works at a boarding school in…
Rhiley Fitch just turned five years old on May 10th. She is diagnosed with a condition called apraxia, which is a rare motor speech disorder. Rhiley is determined to learn…
Action CIND is a charity which strives to raise awareness for chronic immunological and neurological, or CIND, diseases. The organization was founded by Alison Rae, who lives with myalgic encephalomyelitis…
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