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Despite the Pandemic, 2020 Had More Drug Development for Rare Diseases than 2019

• Post author:Trudy Horsting
• Post published:March 22, 2021
• Post category:Rare Disease

Rare Disease Day is February 28th. This year, it caused a lot of reflection among healthcare professionals and families impacted by rare conditions. How has the pandemic shifted progress in…

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RT001 Granted Rare Pediatric Disease Designation for INAD, FA

• Post author:Jessica Lynn
• Post published:March 22, 2021
• Post category:Friedreich ataxia/Friedreich's Ataxia/Infantile axonal neuropathy

In a press release from late February 2021, biopharmaceutical company Retrotope shared that its lead drug candidate, RT001, received Rare Pediatric Disease designation for the treatment of patients with Friedreich's…

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Study of the Week: Using CRISPR to Investigate Cancer Cell Division

• Post author:James Moore
• Post published:March 22, 2021
• Post category:Cancer

Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…

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“I Have IH” Campaign Raises Awareness on Idiopathic Hypersomnia

• Post author:Jessica Lynn
• Post published:March 22, 2021
• Post category:Idiopathic Hypersomnia

In late March 2021, the Hypersomnia Foundation and biopharmaceutical company Jazz Pharmaceuticals, Inc. ("Jazz") partnered up to launch the "I Have IH" campaign. The campaign is designed to raise awareness…

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Long-Term Data Released on eli-cell, a Cerebral Adrenoleukodystrophy Treatment

• Post author:Kendall Mason
• Post published:March 22, 2021
• Post category:Adrenoleukodystrophy

bluebird bio has created a gene therapy for the treatment of cerebral adrenoleukodystrophy (CALD), which is now making its way through a clinical development program. According to Businesswire, bluebird has…

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Two Groups Provide Funding for Cystinosis Research

• Post author:Kendall Mason
• Post published:March 22, 2021
• Post category:Cystinosis

A new study is in the works, and it will investigate how cystinosis, a genetic disorder characterized by an accumulation of the amino acid cystine in the cells, leads to…

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FDA Rejects Ropeginterferon alfa-2b for Polycythemia Vera

• Post author:Jessica Lynn
• Post published:March 22, 2021
• Post category:Polycythemia vera

The drug development process is not always easy or quick. It consists of drug discovery, preclinical and clinical research, and FDA approval. However, sometimes developers are sent back to the…

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Lupkynis for Lupus Nephritis Has Favorable Cost-Effectiveness

• Post author:Jessica Lynn
• Post published:March 22, 2021
• Post category:Lupus Nephritis

How much should medication cost, and are these treatments cost-effective and accessible for patients? In a press release, biopharmaceutical company Aurinia Pharmaceuticals, Inc. ("Aurinia") shared that an independent review performed…

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SGT-001 Safety and Efficacy Data Available from IGNITE DMD Clinical Trial

• Post author:Jessica Lynn
• Post published:March 22, 2021
• Post category:Duchenne Muscular Dystrophy/Muscular Dystrophy/Timely

Since its inception, life sciences company Solid Biosciences Inc. has been working to develop treatment options for patients with Duchenne muscular dystrophy (DMD). According to a recent press release, the…

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