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…
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…
In 2015, the United States approved Kanuma (sebelipase alfa) for the treatment of infants living with rare lysosomal acid lipase deficiency. However, the therapy was not approved in other countries;…
According to a story from Healio, a recent study has found that use of hydroxyurea in alignment with treatment guidelines has the potential to bring down symptoms of cerebrovascular disease…
According to a story from Healio, Rohit Kohli, MBBS, MS, division chief of gastroenterology, hepatology and nutrition at Children’s Hospital Los Angeles, spoke at the World Congress on Insulin Resistance, Diabetes…
The drug Aficamten is being developed by Cytokinetics as a pill taken once daily for hypertrophic cardiomyopathy (HCM). It is a genetic disease where due to a thickness in the…
In 2023, there have been new drug approvals and increased patient awareness. The American Cancer Society reported a total of 1,958,310 new cancer cases in 2023. Chemotherapy remains the standard…
Carisma Therapeutics, the manufacturer of CT-0508, issued a press release stating that researchers are conducting a combination study using CAR-M cell therapy plus Keytruda (pembrolizumab) in the treatment of overexpressing…
According to a story from Science Daily, a team of scientists affiliated with the Salk Institute recently published a study on December 6, 2023 focused on how a class of…
According to a recent American Society of Clinical Oncology (ASCO) press release, Dr. Jason Westin provided testimony regarding a nationwide shortage of cancer drugs, numbering more than 15 on the…
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