The Sun Sucks, But You Do Not
My whole life growing up, there was this specific buzzing sound that would come alive on the sunniest of days. I’ve been told over the years it’s because of everything…
Continue Reading
The Sun Sucks, But You Do Not
FDA Accelerates PORT-77 for Rare Sunlight-Sensitive Disorders
Portal Therapeutics, in partnership with GondolaBio, has achieved a milestone with the FDA granting orphan drug and fast track status to its oral therapy candidate, PORT-77. This investigational drug, according…
Continue Reading
FDA Accelerates PORT-77 for Rare Sunlight-Sensitive Disorders
Sunlight Brings Pain: A Mother’s Journey to Understand Her Daughter’s Rare Condition
For most families, a sunny day is a source of joy, but for the family of five-year-old Evie-Rose, sunlight brings distress and fear. As was reported in the Scottish Sun,…
Continue Reading
Sunlight Brings Pain: A Mother’s Journey to Understand Her Daughter’s Rare Condition
Alexas_Fotos / Pixabay
These Rare Diseases Cause Pain After Sun Exposure. Dersimelagon Could Help.
Porphyria refers to a group of rare disorders that cause issues with the production of heme, which is part of what makes up hemoglobin (a protein in your red blood…
Continue Reading
These Rare Diseases Cause Pain After Sun Exposure. Dersimelagon Could Help.
source: pixabay.com
Misconceptions About Porphyria Can Make it Even Harder to Handle
Living with a rare disease is difficult for so many reasons. One of the lesser talked about struggles of living with a rare condition is the fact that so few…
Continue Reading
Misconceptions About Porphyria Can Make it Even Harder to Handle
source: pixabay.com
The FDA is Developing, Approving, and Expediting Exciting New Therapies
Janet Woodcock, Director of the FDA’s Center for Drug Evaluation and Research (CDER) recently gave an interview to the publication FDA Voices about the agency’s approval in 2019 of a…
Continue Reading
The FDA is Developing, Approving, and Expediting Exciting New Therapies
The FDA Has Just Approved the Very First Therapy for Erythropoietic Protoporphyria
EPP Erythropoietic protoporphyria (EPP) is a rare disease that causes extreme light sensitivity. This sensitivity is so extreme that being outside is extremely difficult for patients and many only come…
Continue Reading
The FDA Has Just Approved the Very First Therapy for Erythropoietic Protoporphyria
FDA Sets Date for Ruling on EPP New Drug Application
According to CheckOrphan, Clinuvel Pharmaceuticals recently announced that their drug Scenesse has received a Prescription Drug User Fee Act (PDUFA) date from the FDA, set for July 8th of this…
Continue Reading
FDA Sets Date for Ruling on EPP New Drug Application
Source: www.pixabay.com
Fun in the Sun? How One Little Girl Beat the Summer Heat after an EPP Diagnosis
The American Porphyria Foundation has a section on its website dedicated to telling patient stories. It gives voice to people's experiences and allows them to connect with others and share…
Continue Reading
Fun in the Sun? How One Little Girl Beat the Summer Heat after an EPP Diagnosis
