Mary Chapman, features writer —

If you have porphyria, you likely may regularly have trouble getting a good night’s sleep. There are ways to help remedy that. What is porphyria? Porphyria refers to a group of disorders in which affected individuals cannot make hemoglobin, the protein that binds oxygen in…

If you have acute porphyria, it’s important to know that diet and nutrition may help to ease your symptoms. What is porphyria? Porphyria refers to a group of disorders in which affected individuals cannot make hemoglobin, the protein that binds oxygen in red blood cells. Due to…

The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. Survey questions cover a patient’s physical and mental health, supply of treatments, and access to healthcare, among other…

The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. Called the NORD COVID-19 Critical Relief Program, the effort will provide up to $1,000 annually to those eligible to…

To help patients and families facing out-of-pocket medical costs, The Assistance Fund (TAF) has opened a new program for eligible individuals living with porphyria. The TAF Porphyria Financial Assistance Program helps with therapy-related copayments, health insurance premiums, and incidental medical expenses related to the metabolic disorder, which has…

Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, and aims to supports work by researchers and clinicians into a better understanding of and treatments for rare diseases, as well…

An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…

Alnylam Pharmaceuticals has tapped PANTHERx Rare Pharmacy to be a limited distribution partner for its newly approved therapy Givlaari (givosiran) to treat acute hepatic porphyria (AHP). The medication was approved in November by the U.S. Food and Drug Administration (FDA) for adults with AHP, a…

In an ongoing effort to help advocacy groups address unmet needs in rare diseases such as porphyria, therapeutic company Alnylam is calling for applicants for its second annual global grants program, called Advocacy for Impact. The $50,000 grants will fund new projects that focus specifically on…

Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions of people — estimates run as high as 350 million — they are thought to impact. Across countries, patients, caregivers and advocates will paint faces, wear…