Funding Goal Achieved for Pulmonary Arterial Hypertension Clinical Trial
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Funding Goal Achieved for Pulmonary Arterial Hypertension Clinical Trial

According to a story from Euroinvestor, the drug development company Resverlogix Corp. recently announced that it has received the necessary $2.9 million in funding for a Phase 2 clinical trial…

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The Argument for Mandatory Newborn Screening and Government Subsidies for Rare Diseases in India

The State of Rare Disease in India There are 7,000 known rare diseases worldwide. The Organization for Rare Diseases India (ORDI) estimates 70 million people are affected by a rare diagnosis…

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A Recap on 2018: Record Number of Drugs Approved by the FDA for Rare Diseases

Each year the Food and Drug Administration approves dozens of new therapies to fulfill unmet needs for various patient populations. It's just been published that in 2018 they approved a…

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This Just In: India Never Implemented their Rare Disease Policy as Promised
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This Just In: India Never Implemented their Rare Disease Policy as Promised

We all got really excited last year when India implemented a new plan to benefit rare disease patients. It was called the National Rare Disease Policy. Basically, it put 12.86…

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FDA Commissioner Releases Statement on Targeted Therapies, New Efficiency Measures, and Modernizing Development
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FDA Commissioner Releases Statement on Targeted Therapies, New Efficiency Measures, and Modernizing Development

According to a story from Pharma Voice, Dr. Scott Gottlieb, the current commissioner of the US Food and Drug Administration (FDA) has recently released a statement in regards to efforts…

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Program Aims to Help Diagnose Certain Rare Diseases More Quickly
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Program Aims to Help Diagnose Certain Rare Diseases More Quickly

According to a story from PR Newswire, a new program called The Lantern Project is working to provide a free testing program that allow doctors to more quickly diagnose patients…

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New Updates on Patients Taking Part in Studies of a Gene Therapy for Fabry Disease Have Been Released
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New Updates on Patients Taking Part in Studies of a Gene Therapy for Fabry Disease Have Been Released

Avrobio has released updates on patients in two clinical trials of a potential gene therapy for Fabry disease. According to the announcement, two patients taking part in a Phase 1…

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This Dad Climbed a Literal Mountain for His Daughter with Pompe Disease

The Malaysia Lysosomal Diseases Association (MLDA) was formed in 2011 by the hands of eight families. Their goal was to raise awareness and funds for those diagnosed with lysosomal storage…

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So Why DOES Canada have Scarcer Access to Rare Disease Therapies?
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So Why DOES Canada have Scarcer Access to Rare Disease Therapies?

According to a story from CheckOrphan, Durhane Wong-Rieger, the CEO of the Canadian Organization for Rare Disorders, says that the Canadian health system has been letting down the roughly three…

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The FDA Just Approved the New Drug Application for Migalastat, a Treatment For Fabry Disease
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The FDA Just Approved the New Drug Application for Migalastat, a Treatment For Fabry Disease

According to an article from globenewswire.com, the pharmaceutical company Amicus Therapeutics announced that the Food and Drug Administration (FDA) has the company's New Drug Application for migalastat with filing for…

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