Rates of colorectal cancer are on the rise. In the US and other parts of the developed world, people seem to be developing this cancer earlier in life, for reasons…
A study led by a team of German scientists provides evidence of the safety and efficacy of CD19 CAR T cell therapy and its impact on autoimmune diseases. The study…
According to a story from Healio, encouraging results from a phase 2 trial could herald a new standard of treatment for metabolic dysfunction-associated steatohepatitis (MASH). The investigational therapy is called…
The European Medicines Agency (EMA) explains that monepantel is an anthelmintic therapy, or a type of therapy that is used to expel worm-like parasites called helminths or nematodes from the gut.…
According to a story from Medscape, a new set of guidelines from the Kidney Disease: Improving Global Outcomes (KDIGO) aims to improve and update standards of care for antineutrophilic cytoplasmic…
According to a story from BioSpace, the current standard approaches for drug development aren't well suited for rare diseases, which have unique characteristics in comparison to more widespread illnesses. As…
At the end of January 2024, global healthcare leader Eli Lilly and Company shared that its therapy OLUMIANT (baricitinib) received regulatory approval in Canada for the treatment of severe alopecia…
People living with blood disorders like sickle cell disease or thalassemia will now have access to a new blood test that will reduce transfusion side effects. England’s National Health Service…
For years, scientists have been working on a completely new concept that generates new neurons in mice with Huntington's disease. They were able to demonstrate that the new cells could…
The 17th Annual Congress of the European Association for Haemophilia and Allied Disorders took place this year from February 6-9, 2024. During the Congress, stakeholders came together to discuss how…
Imagine if you had recurring attacks of severe and unexplained swelling beneath your skin. For individuals living with hereditary angioedema (HAE), a rare inherited disorder, this isn't just a mere…
The parents had brought their baby girl home only four days ago when they received an urgent call from the hospital asking them to bring the baby right back for…
Acknowledgment: This patient story is sponsored by Ipsen Biopharmaceuticals, Inc. and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing…
Prilenia Therapeutics, Waltham, Massachusetts and Naarden, Netherlands is a biotechnology company with a focus on developing therapeutics to treat neurodevelopmental disorders and neurodegenerative diseases. Prilenia presented data this month at…
Written by Maria Picone, TREND Community Ten years ago, my daughter was born with a rare, complex disease known as Prader-Willi syndrome (PWS). PWS is best known for causing insatiable…
The primary outcome from the Phase III NETTER-2 clinical trial as reported recently in Globe Newswire, was heralded as extending progression free survival from 8.5 months to 22.8. The participants…
According to reporting from Healio Psoriatic Disease, the Food and Drug Administration (FDA) recently updated the label for Dupixent (dupilumab) in the indication of moderate-to-severe atopic dermatitis with moderate-to-severe hand…
In January 2024, the FDA approved Hyqvia (Immune Globulin Infusion 10% [Human]) as a maintenance therapy for adults living with chronic inflammatory demyelinating polyneuropathy (CIDP), a rare neurological disorder. As…
The Griffins of Harrisburg, North Carolina enrolled their son in the AGAMREE clinical trial in 2016. Their son was the first participant in the trial studying the investigational drug vamorolone…
Sarepta Therapeutics’ treatment Elevidys (delandistrogene moxeparvovec-rokl) received conditional approval from the FDA in June 2023. The designation implies that if the drug is used in accordance with its label, it…
According to an article published in Healthcare Dive, a recent study published in the academic journal JAMA indicated that patients receiving treatment in hospitals owned by private equity firms were at an…
The immune system is constantly on the alert for cells that have sustained DNA damage. When a cell divides its DNA molecules are, to varying degrees, usually damaged. Yet researchers…
The agency in Guangzhou, China, that arranged three-year-old Shelby’s adoption by a family in the US disclosed to Michelle and Adam Campbell that Shelby had beta thalassemia disorder. Yet, that…
Eliza is now 13 years old and was diagnosed at the age of three with a neurodegenerative disorder known as Sanfilippo syndrome. Eliza’s mother, Dr. Cara O’Neill, a pediatrician and…
In December 2023, clinical-stage biopharmaceutical company Clene Nanomedicine ("Clene") shared that it had met with the U.S. Food and Drug Administration (FDA) regarding Accelerated Approval for CNM-Au8®, an experimental therapy…
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