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July 17, 2020July 17, 2020

Antivirals Seen to Prevent Porphyria Cutanea Tarda in Hepatitis C Patients

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Antiviral treatment can prevent the onset of porphyria cutanea tarda (PCT) in people with hepatitis C and urine abnormalities indicating this form of porphyria, a study found. The study, “Resolution of ... Read more

July 13, 2020July 13, 2020

National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US

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The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease ... Read more

July 10, 2020July 10, 2020

Long-term Givlaari Treatment Greatly Lowers AIP Attacks, Study Shows

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People with acute intermittent porphyria (AIP) who were treated with Alnylam Pharmaceuticals’ RNA-based therapy Givlaari (givosiran) experienced a rapid and lasting reduction in porphyria attacks — and most remained attack-free for ... Read more

June 22, 2020June 22, 2020

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

cystic fibrosis, News, syndicated

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will ... Read more

June 19, 2020June 19, 2020

American Porphyria Foundation Among Winners in Alnylam’s ‘Advocacy for Impact’ Grants

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Alnylam Pharmaceuticals has awarded a total of $270,000 to seven patient advocacy groups, including the American Porphyria Foundation (APF), in the company’s Advocacy for Impact grants program. The second annual program, designed to ... Read more

June 12, 2020June 12, 2020

Givlaari Markedly Reduces AIP Attacks, Phase 3 Trial Shows

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People with acute intermittent porphyria (AIP) who were treated with Alnylam Pharmaceuticals’ RNA-based therapy Givlaari (givosiran) had a 74% reduction in porphyria attacks and sustained decreases in toxic products ... Read more

June 9, 2020June 10, 2020

Coalition Will Address Racial Disparities in Rare Disease Communities

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The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis ... Read more

June 5, 2020June 5, 2020

Recruiting Underway for New Study Testing Light Exposure Device for EPP

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A new study testing a light exposure measuring device designed to help people with erythropoietic protoporphyria (EPP) better manage their symptoms is recruiting participants. A total of 30 EPP ... Read more

May 29, 2020June 1, 2020

MT-7117 Poised for Phase 3 Trial in Two Types of Porphyria

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A Phase 3 clinical trial of Mitsubishi Tanabe’s investigational therapy MT-7117 (dersimelagon) for people with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP) has been launched. The study (NCT04402489) will take ... Read more

May 28, 2020May 28, 2020

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

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While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene ... Read more

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