Editor’s Choice: So Many Rare Patient Stories, So Little Time
Hey...Is That Autumn on the Horizon? Well...kind of but sort of no, it is still pretty hot where we are; but it is only a matter of time before we…
Hey...Is That Autumn on the Horizon? Well...kind of but sort of no, it is still pretty hot where we are; but it is only a matter of time before we…
According to a story from curetoday.com, cancer patient registries have had a major impact in improving outcomes for patients. Take the story of Rhonda and Jeff Meckstroth as an example.…
As reported in Cystic Fibrosis News Today, a new drug combination created for Cystic Fibrosis, a mix of Vertex's elexacaftor, tezacaftor, and Kalydeco has been granted priority review. The new…
Originally reported in People Magazine, a family struggled for years to figure out what was happening to their son, Cohen Bramlee, who for years received his nutrients from a central…
According to a story from The Rheumatologist, a rare complication linked to the monoclonal antibody drug rituximab called rituximab-induced serum sickness (RISS) appears when patients using the drug have immune…
Last week, we spotlighted a New York Times story about how under the Trump Administration’s new immigration policy, six migrants undergoing lifesaving treatments would be abruptly deported; including a young woman…
According to a story from globenewswire.com, the biopharmaceutical company Processa Pharmaceuticals, Inc., has recently announced that it has completed enrollment for its phase 2 clinical trial. This trial will be…
According to a recent publication from Moneycontrol, patient advocacy groups in India are calling for the country's Minister of Health and Family Welfare, Harsh Vardhan, to establish a provisional system…
According to a publication from the Korea Times, the Eone Diagnomics Genome Center in Incheon, South Korea, is working to improve patients' accessibility to their own genetic sequence. Researchers believe…
According to a publication from The Inside Press, researchers at the Burke Neurological Institute in White Plains, New York recently conducted a clinical trial focused on the potential use of…
According to a story from news-medical.net, scientists may have made a significant breakthrough discovery that could make cancer treatment more effective. The discovery involves a new method that breaks through…
According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Rhythm Pharmaceuticals, Inc., has recently announced the results from two phase 3 clinical trials. These trials were testing the company's experimental…
According to a press release from Mateon Therapeutics, Mateon's recent merger agreement with PointR Data establishes plans to combine the use of artificial intelligence and data collection for the development…
According to a story from New Statesman, the last month has seen a number of new drugs introduced that could have a significant impact on several dangerous tropical diseases, such…
According to a story from BioSpace, the biopharmaceutical company CrystalGenomics, Inc. recently announced that the US Food and Drug Administration (FDA) has granted the company's investigational drug candidate CG-745 Orphan…
According to a story from NBC News, the death of a patient following a fecal transplant procedure has finally prompted the US Food and Drug Administration (FDA) to take more…
As my children are getting older they seem to create elaborate stories of why they didn’t clean up their room or complete any other task on their to-do list. I’ve…
According to a story from the National Center for Advancing Translational Sciences (NCATS), research supported by the center is making it possible for kids born with serious, rare genetic disorders…
Netflix debuted a documentary series this month called 'Diagnosis' -- based on a New York Times Magazine column -- which follows individuals with or caregiving for patients with a mysterious,…
According to a press release from Eiger Biopharmaceuticals, the American Food and Drug Administration (FDA) recently granted the Company's experimental hepatitis delta virus drug (hepatitis D), peginterferon lambda, breakthrough therapy…
Aevolve, a medical innovation platform, announced last week that it will be making rare disease research a top priority in its blockchain-powered accelerator, which provides a pathway for alternative funding…
It was announced late last week that Justice Ruth Bader Ginsburg had just completed three weeks of radiation treatment for tumors on her pancreas. Pancreatic cancer occurs in the pancreas, an organ…
According to a press release from Synlogic Therapeutics, the Company recently halted development of its investigational hyperammonemia treatment SYNB1020 after an unsuccessful phase 1/2 clinical study. About Hyperammonemia Hyperammonemia is…
Glioblastoma Glioblastoma is a rare type of brain cancer. Sadly, it is the most aggressive form. The median life expectancy for the disease is between 11 and 15 months and…