New FDA Regulation on Laboratory Developed Tests Draws Objections from Healthcare Groups
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New FDA Regulation on Laboratory Developed Tests Draws Objections from Healthcare Groups

As reported in Biopharma Dive, the FDA’s decision to strengthen its authority involving laboratory developed tests (LDTs) has drawn considerable objections from various healthcare groups. However, the Agency has held…

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RETINA WORLD CONGRESS: Diabetic Macular Edema Clinical Trials Lack Patient Diversity

When it comes to medical research, diversity, equity, and inclusion need to be larger parts of the conversation. As it stands now, medical research can be fiercely hierarchical, expensive, and homogenous in terms of…

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Wild Wickenburg Bull Riding Fundraising Raises $5K for Teen with MPGN II

In Wickenburg, Arizona, the community sticks together. Community means strength.  So when Noah Ahnlund was diagnosed with a rare autoimmune disease called membranoproliferative glomerulonephritis type II (MPGN II/dense deposit disease)…

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AOC 1001 Earns Breakthrough Therapy Designation for Myotonic Dystrophy Type 1 (DM1)
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AOC 1001 Earns Breakthrough Therapy Designation for Myotonic Dystrophy Type 1 (DM1)

Currently, there are no approved treatment options for myotonic dystrophy type 1 (DM1), a progressive neuromuscular disease. As the disease progresses, affected individuals experience muscle weakness, respiratory distress, and cardiac…

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New Podcast Episode: The Importance of IPF Clinical Trials, feat. Patient Advocate Murray Walz
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New Podcast Episode: The Importance of IPF Clinical Trials, feat. Patient Advocate Murray Walz

Patient Worthy's award-winning podcast "Wait, How Do You Spell That? A Rare Disease Podcast" is back with a new episode. This week, Colby is sitting down with Murray Walz, a…

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STXBP1 Disorders Foundation President Charlene Son Rigby on the First White House Rare Disease Forum
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STXBP1 Disorders Foundation President Charlene Son Rigby on the First White House Rare Disease Forum

On February 28th, 2024, the first-ever White House Rare Disease Forum took place. Hosted by the White House Office of Science and Technology Policy, this forum provided a platform for…

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ICYMI: INZ-701 Begets Positive Data in Clinical Study on ABCC6 Deficiency and ENPP1 Deficiency
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ICYMI: INZ-701 Begets Positive Data in Clinical Study on ABCC6 Deficiency and ENPP1 Deficiency

Biopharmaceutical company Inozyme Pharma ("Inozyme") has become a leader at the forefront of developing innovative therapies for rare diseases such as ENPP1 deficiency (the childhood form is known as generalized…

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