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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

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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

May 25, 2020May 25, 2020

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

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European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session ... Read more

May 22, 2020May 22, 2020

Low Blood Sodium of Porphyria May Also Cause Reversible Brain Lesions

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The low blood sodium levels associated with acute intermittent porphyria (AIP) may also contribute to reversible splenial lesion syndrome (RESLES), a case report suggests. The study, “Reversible splenial lesion syndrome ... Read more

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