Clinical Trial of MGTA-456 Looks Promising for Inherited Metabolic Disorders
source: pixabay.com

Clinical Trial of MGTA-456 Looks Promising for Inherited Metabolic Disorders

  According to an article in BioPortfolio, Magenta Therapeutics, a biotechnology company based in Cambridge, Mass., recently announced updates to its Phase II trial of MGTA-456 for the treatment of…

Continue Reading Clinical Trial of MGTA-456 Looks Promising for Inherited Metabolic Disorders
India Has Introduced a New Rare Disease Policy Draft, But it Doesn’t Benefit all Rare Patients
source: pixabay.com

India Has Introduced a New Rare Disease Policy Draft, But it Doesn’t Benefit all Rare Patients

India first drafted a national rare disease policy back in 2017. Unfortunately, it was never enacted due to budgeting and implementation issues. This draft included suggestions such as a corpus…

Continue Reading India Has Introduced a New Rare Disease Policy Draft, But it Doesn’t Benefit all Rare Patients
A Proof-of-Concept Study for an Experimental Hurler Syndrome Treatment is Going Well so Far
sasint / Pixabay

A Proof-of-Concept Study for an Experimental Hurler Syndrome Treatment is Going Well so Far

According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Orchard Therapeutics recently announced the release of some data from an ongoing proof-of-concept study. This special trial is testing the company's…

Continue Reading A Proof-of-Concept Study for an Experimental Hurler Syndrome Treatment is Going Well so Far

Orchard Therapeutics Acquires Global Licensing Agreement for Experimental Mucopolysaccharidosis Drug

According to a publication from Biospace, British clinical drug developer Orchard Therapeutics has secured global intellectual property rights to research, manufacture, and market a gene therapy program for the treatment…

Continue Reading Orchard Therapeutics Acquires Global Licensing Agreement for Experimental Mucopolysaccharidosis Drug

Rare Diseases Could Be Diagnosed Earlier with Better Training for GPs to Avoid Years of Misdiagnosis

Although each disease on its own may be rare, a huge number of people are living with a rare disease-- and half of these people are children. About 30 percent of these children…

Continue Reading Rare Diseases Could Be Diagnosed Earlier with Better Training for GPs to Avoid Years of Misdiagnosis

This Gene Editing Approach Could Help Treat Genetically Linked Rare Diseases

According to a story from medicalxpress.com, a team of scientists at the University of Massachusetts Medical School have successfully developed a strategy for editing genes that could be used to…

Continue Reading This Gene Editing Approach Could Help Treat Genetically Linked Rare Diseases
Patients Are Being Asked for Their Opinions on Treatments For Lysosomal Storage Diseases
Source: Pixabay

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

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…

Continue Reading Patients Are Being Asked for Their Opinions on Treatments For Lysosomal Storage Diseases
Close Menu