Progeria
New Treatment Could Improve the Life Expectancy for People with Progeria
There have been extreme improvements in genetic engineering over the past fifty years since it was pioneered in the 1970s. It has been used
Rare Patient News. Well Done.
Presentation Highlights Need for Improvements in Familial Hypercholesterolemia Care
- November 21, 2019
FDA Gives Rare Pediatric Disease Designation to Experimental Drug for Treating Rett Syndrome
Study Reveals New Approach for Diagnosing Nonalcoholic Steatohepatitis
A New Treatment for Cushing’s Syndrome Nears EU Approval
LATEST PATIENT WORTHY ARTICLES
Neurofibromatosis
This Mom Raising a Son with Neurofibromatosis Started a Foundation to Support Parents
According to a story from Soweto Urban, mother Lebohang Leuta is no stranger to the challenges that come with raising and taking care of a
Idiopathic Pulmonary Fibrosis
A Recent Study from Yale Tracks the Progression of Scarring in Idiopathic Pulmonary Fibrosis
According to a story from EurekAlert!, a collaborative study headed by researchers from Yale University has furthered the understanding of the mechanisms and progression of
Angelman Syndrome
These Two Companies Just Announced a Partnership to Develop Multiple Gene Therapies
According to a story from BioSpace, the gene therapy companies Sarepta Therapeutics, Inc. and StrideBio, Inc. have recently announced the completion of a licensing and
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Hereditary ATTR Amyloidosis
ICYMI: hATTR Amyloidosis was Untreatable Until a Gene Silencing Drug was Approved in the UK
An article appeared recently in BioNews announcing the first-ever approval of an RNA-based therapy for use on the British National Health Service. The name
Rare Disease
Teen Gets the First Ever Double Lung Transplant Due to Vaping Associated Lung Injury
Cigarettes have been repackaged for the 21st century. The new millennial pass-time is vaping, an electronic device that heats a liquefied solution of nicotine or
UPCOMING EVENTS
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Why Are Adults in the US Having Trouble Accessing Their Medications?
Modern medication has the ability to save lives and improve living standards. However, medicine is less beneficial when patients’ prescribed treatments are delayed, and entirely
PW Collaborator
November 19, 2019
