Hunter Syndrome – Page 2

Experimental Treatment for Hunter Syndrome Earns Rare Pediatric Disease Designation and Orphan Drug Designation

Post author:James Moore
Post published:June 20, 2019
Post category:Hunter Syndrome MPS II Mucopolysaccharidosis

According to a story from globenewswire.com, the biopharmaceutical company Denali Therapeutics, Inc. has recently announced that its experimental product candidate DNL310, which is currently being developed as a treatment for…

12019 / Pixabay

Intern with Hunter Syndrome Draws Career Inspiration from Patient Experiences

Post author:James Moore
Post published:May 15, 2019
Post category:Hunter Syndrome

According to a story from Palomar Health, a patient with Hunter syndrome is about to finish up the company's Pathmaker Internship program. The patient in question is Kyle Underwood, who…

source: pixabay.com

India’s Policy for Rare Disease Treatment Mired in Legal Gridlock

Post author:Scott Carlson
Post published:May 1, 2019
Post category:Hunter Syndrome Rare Disease

An Indian health policy initiative meant to provide funding to rare disease patients has struggled to gain approval from the country's administration, according to a publication from DNA India. First…

The Argument for Mandatory Newborn Screening and Government Subsidies for Rare Diseases in India

Post author:Trudy Horsting
Post published:March 8, 2019
Post category:Fabry Disease Gaucher Disease Hunter Syndrome

The State of Rare Disease in India There are 7,000 known rare diseases worldwide. The Organization for Rare Diseases India (ORDI) estimates 70 million people are affected by a rare diagnosis…

Slow but Steady Progress Toward Gene Editing for Hunter Syndrome and Hurler Syndrome

Post author:Trudy Horsting
Post published:February 14, 2019
Post category:Hunter Syndrome Hurler Syndrome

Gene Editing What is gene editing? It's been a hot topic over the last few years, but basically the concept is this: instead of disabling a malfunctioning gene, researchers are…

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Patient Centricity Helps Facilitate The Introduction of New Therapies for Rare Diseases

Post author:James Moore
Post published:December 31, 2018
Post category:Cystic Fibrosis Familial Hypercholesterolemia Hunter Syndrome Rare Disease

According to a story from Pharmaphorum, there are a number of new treatments that have been introduced or have seen major strides in development over the last year. More patient…

PixLoger / Pixabay

In-Body Genome Editing: A New Potential Cure for Hunter Syndrome

Post author:Samuel Sachs
Post published:September 19, 2018
Post category:Hunter Syndrome MPS II Rare Disease

According to a report by CheckOrphan, a recent study investigated the use of genome editing directly in the body. The research involved the use of these editing techniques in cases…

lisichik / Pixabay

Preliminary Results of Gene Editing Experiment Suggest Potential for Treating Hunter Syndrome

Post author:James Moore
Post published:September 7, 2018
Post category:Hunter Syndrome Rare Disease

According to a story from the Santa Cruz Sentinel, the earliest results from a critical gene editing study could offer some encouragement for those hoping to treat rare genetic diseases…

lil_foot_ / Pixabay

Family of a Boy With Hunter Syndrome is Racing Against Time

Post author:James Moore
Post published:August 3, 2018
Post category:Hunter Syndrome MPS II (Hunter Syndrome)Mucopolysaccharidosis

According to a story from philly.com, four year old Ethan Lieber of the Roxborough neighborhood in Philadelphia is one of just 500 people in the US with Hunter syndrome. This…

source: pixabay.com

A Mother’s Fundraising Campaign Could be Critical For Finding a Cure For Hunter Syndrome

Post author:James Moore
Post published:May 12, 2018
Post category:Hunter Syndrome MPS II

According to a story from The Tennessean, mother Melissa Hogan has been inspired by her son Case's Hunter syndrome to start a nonprofit organization called Project Alive. The primary purpose…

Source: Pixabay

Patients Are Being Asked for Their Opinions on Treatments For Lysosomal Storage Diseases

Post author:Anna Hewitt
Post published:April 13, 2018
Post category:Gaucher Disease Hunter Syndrome Hurler Syndrome Lysosomal Disease Pompe Disease

In an effort to better understand patient experiences, researchers at the Massachusetts General Hospital are surveying people with lysosomal storage diseases about how they view their treatments, reports CheckOrphan. The…

Experimental Treatment for Hunter Syndrome Earns Rare Pediatric Disease Designation and Orphan Drug Designation

Intern with Hunter Syndrome Draws Career Inspiration from Patient Experiences

India’s Policy for Rare Disease Treatment Mired in Legal Gridlock

The Argument for Mandatory Newborn Screening and Government Subsidies for Rare Diseases in India

Slow but Steady Progress Toward Gene Editing for Hunter Syndrome and Hurler Syndrome

Patient Centricity Helps Facilitate The Introduction of New Therapies for Rare Diseases

In-Body Genome Editing: A New Potential Cure for Hunter Syndrome

Preliminary Results of Gene Editing Experiment Suggest Potential for Treating Hunter Syndrome

Family of a Boy With Hunter Syndrome is Racing Against Time

A Mother’s Fundraising Campaign Could be Critical For Finding a Cure For Hunter Syndrome

Patients Are Being Asked for Their Opinions on Treatments For Lysosomal Storage Diseases

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