Kate Middleton Visits Boys with Hunter Syndrome and Dystonic Cerebral Palsy

  East Anglia's Children's Hospices in Norfolk, U.K., has recently opened up its new facilities after renovations. To celebrate this opening, Kate Middleton, the Duchess of Cambridge, visited and spoke…

Continue Reading Kate Middleton Visits Boys with Hunter Syndrome and Dystonic Cerebral Palsy
Company Seeks to Treat Hunter Syndrome (MPS II) in China With New Drug Application
liaoyiye / Pixabay

Company Seeks to Treat Hunter Syndrome (MPS II) in China With New Drug Application

According to a story from BioSpace, the biopharmaceutical company CANBridge Pharmaceuticals announced that it has recently submitted a New Drug Application with China's National Medicinal Products Administration (NMPA) for its…

Continue Reading Company Seeks to Treat Hunter Syndrome (MPS II) in China With New Drug Application
India Turns to Enzyme Replacement Therapy as it Seeks to Address Rare Diseases
klbz / Pixabay

India Turns to Enzyme Replacement Therapy as it Seeks to Address Rare Diseases

According to a story from health.economictimes.indiatimes.com, the country of India is currently beginning to take more steps in order to address the dire unmet medical need of its rare disease…

Continue Reading India Turns to Enzyme Replacement Therapy as it Seeks to Address Rare Diseases
Takeda Pharmaceutical Launches Trio of Enzyme Replacement Therapies in India to Treat Lysosomal Storage Disorders
source: pixabay.com

Takeda Pharmaceutical Launches Trio of Enzyme Replacement Therapies in India to Treat Lysosomal Storage Disorders

According to a press release from Takeda Pharmaceutical Company published by For Press Release, the Company has launched a collection of enzyme replacement therapies for use in the treatment of…

Continue Reading Takeda Pharmaceutical Launches Trio of Enzyme Replacement Therapies in India to Treat Lysosomal Storage Disorders

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

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…

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

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

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…

Continue Reading The Argument for Mandatory Newborn Screening and Government Subsidies for Rare Diseases in India
Patient Centricity Helps Facilitate The Introduction of New Therapies for Rare Diseases
stevepb / Pixabay

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

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…

Continue Reading Patient Centricity Helps Facilitate The Introduction of New Therapies for Rare Diseases
Preliminary Results of Gene Editing Experiment Suggest Potential for Treating Hunter Syndrome
lisichik / Pixabay

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

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…

Continue Reading Preliminary Results of Gene Editing Experiment Suggest Potential for Treating Hunter Syndrome
A Mother’s Fundraising Campaign Could be Critical For Finding a Cure For Hunter Syndrome
source: pixabay.com

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

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…

Continue Reading 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
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