Our partner, the MDS Foundation, is sponsoring another free event for patients and caregivers. Event Summary: Whether you are a newly diagnosed patient, a long-term survivor, or a caregiver this…
The survival rates for infants and children with brain and spinal cord cancer has improved significantly over the past decades. However, a study published online in early August of…
According to a story from Pharma Times, four new medications were recently approved for coverage on the Scottish NHS by the Scottish Medicines Consortium (SMC). Some of these drugs are…
Nitu is eleven years old. She lives in Shayestanagor in Habiganj (a region within Bangladesh). While most 11-year-old children are full of energy, Nitu must stay at home. Nitu lives…
The US FDA has awarded Breakthrough Therapy Designation to encorafenib (braftoviTM) combined with binimetinib (mektovi®) and cetuximab for use as an investigational (not FDA-approved) treatment of BRAFV600E–mutant metastatic colorectal cancer.…
According to ASHA.org, when Tiffany Turner graduated, she set out to expand opportunities for fellow speech language pathologists like herself. Turner saw a great need in her community for…
Gaucher patients in New Zealand are facing a change. New Zealand’s Pharmaceutical Management Agency (PHARMAC) recently proposed a change in their supported Gaucher treatment from Cerezyme to Elelyso. The switch…
According to VeroNews, one of the biggest issues at the retirement community Treasure Coast is malnutrition. For patients with other serious conditions, like chronic kidney disease, the hospital as…
According to a story from Live Science, a species of tick that is originally found in Asia has begun to appear along the East Coast of the US. The parasite…
A Biologics License Application for ElzonrisTM (also known as tagraxofusp and SL-401) for the treatment of blastic plasmacytoid dendritic cell neoplasm has been accepted for filing by the United States FDA.…
Calling all members of the GNE Myopathy community! The Neuromuscular Disease Foundation (NDF) is hosting its 5th Annual Symposium on GNE Myopathy this August 30-31st at the University of California, Los Angeles. For…
Researchers are exploring a new diagnostic method that could be applied to newborn screening for certain inherited diseases and monitoring of HIV patients. For more detailed information, you can click…
According to DAIC, Akcea Therapeutics Inc., has recently announced their publication of results from their study analyzing patient-to-patient connectivity towards the management of the rare disease familial chylomicronemia syndrome (FCS).…
A gene therapy treatment is being used to treat X-linked severe combined immunodeficiency (X-SCID). Mustang Bio has licensed the treatment from St. Jude Children's Research Hospital. X-SCID is also known…
According to a story from stuff.co.nz, a baby from Auckland has been diagnosed with two extremely rare disorders that only occur in one in every 10 million people. Her name…
According to a story from Science Alert, the human body is a grand and complex natural structure. While it is easy to think of the body as a single entity,…
9% Copyscape An investigational drug called CLR 131 has received Rare Pediatric Disease Designation from the US FDA for the treatment of Ewing’s sarcoma. The source press release can be…
According to a story from wday.com, Paisley Forsell was what you might call a bit of an adrenaline junkie. She likes to live a high energy, active lifestyle. She loves…
According to information provided by the National Hemophilia Foundation, people living with hemophilia now have the opportunity for a greater level of financial support. This new form of aid comes…
A research team led by scientists from the Beth Israel Deaconess Medical Center (BIDMC) have found that arsenic, in combination with the drug all-trans retinoic acid (ATRA), may have the potential…
According to a story from MD Magazine, the US Food and Drug Administration (FDA) has approved the medication migalastat, also known as Galafold, for the treatment of Fabry disease. This…
According to HSC News, researchers at the University of Southern California have engineered a device that may have a sizable impact on those with pediatric hydrocephalus. By troubleshooting the issues…
The award-winning actor Alan Alda has shared his diagnosis of Parkinson’s disease during an interview on the CBS This Morning show. The source video of the interview can be found here on…
A recently published study in the Journal of Biosciences and Medicines has concluded that orthoses may help to improve the gait and balance of patients with Charcot-Marie-Tooth disease. Orthoses are…
A recent study into Periodic Fever, Aphtous stomatitis, Pharyngitis and Adenitis (PFAPA) syndrome investigated the impact of the condition on children and found that is was associated with a reduction…
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