News

January 20, 2021January 20, 2021

NORD’s Caregiver Respite Program Continues Through Pandemic

News, syndicated

Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization ... Read more

January 15, 2021January 15, 2021

Hemin Pretreatment Helped Treat Bacterial Infection in AIP Patient

News

Pretreatment with hemin helped treat a bacterial Helicobacter pylori (H. pylori) infection in a woman with acute intermittent porphyria (AIP), a Japanese case study reports. The treatment protocol outlined in the report ... Read more

January 11, 2021January 11, 2021

NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

News, syndicated

The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD ... Read more

January 8, 2021January 8, 2021

Nerve, Kidney Damage Found to Predict AIP Liver Transplant Outcomes

News

A liver transplant is effective and is associated with high survival rates in people with acute intermittent porphyria (AIP), a study reports. However, severe neuropathy, or nerve damage, and ... Read more

December 18, 2020December 18, 2020

Sun Exposure Damages Skin-Derived Stem Cells in Porphyria Patient

News

Sun exposure may lower the number and affect the properties of skin-derived stem cells in people with variegate porphyria, particularly their ability to give rise to other cell types, ... Read more

December 11, 2020December 11, 2020

AIP Patient With Kidney Disease Improves After Transplant, Case Report Shows

News

A combined liver and kidney transplant can be an effective treatment for acute intermittent porphyria (AIP) patients with kidney disease, a case report shows. The report, “Combined Liver and ... Read more

December 4, 2020December 4, 2020

Determining Normal Urinary Biomarkers Sets Limits for Accurate Porphyria Diagnosis

News

Establishing normal urinary levels of delta-aminolevulinic acid (ALA) and porphobilinogen (PBG) in healthy individuals helps identify people with acute hepatic porphyria (AHP) with high accuracy, a study shows. These reference ... Read more

December 3, 2020December 3, 2020

NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

News, syndicated

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders ... Read more

November 26, 2020November 26, 2020

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

News, syndicated

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to ... Read more

November 20, 2020November 25, 2020

Panhematin for AIP Deemed Essential Medicine By FDA

News

Panhematin (hemin for injection), an approved medication to treat acute intermittent porphyria (AIP), has been deemed an essential medicine by the U.S. Food and Drug Administration (FDA), a designation ... Read more