The CheckRare Brief

A weekly roundup of the latest news in rare disease. The CheckRare Brief delivers a concise roundup of the week's most important developments in rare diseases. Each episode covers FDA approvals, clinical trial updates, conference highlights, scientific publications, and industry news that matter to healthcare professionals, researchers, advocates, and the rare disease community. Produced by CheckRare, The CheckRare Brief helps you stay informed—in just a few minutes each week.

Episodes

Ultragenyx Sells PRV for $210M | The CheckRare Brief Ep 11

October 14, 2026 • 8 mins

In this episode, we discuss Ultragenyx’s Priority Review Voucher that sold for $210 million, the efficacy of newborn screening on MLD, and ethical considerations needed to design and conduct clinical trials.

This past week, Ultragenyx sold its Priority Review Voucher for $210 million.
The company received a PRV for the approval of their glycogen storage disease type Ia treatment, which was approved earlier thi...

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Therapies for MCT8 Deficiency and Nephrotic Syndrome Get FDA Approval

October 8, 2026 • 9 mins

On September 28th, the FDA approved Emcitate, or tiratricol, from Egetis Therapeutics. It's the first-ever treatment for MCT8 deficiency, a condition also known as Allan-Herndon-Dudley syndrome.

MCT8 deficiency is a rare genetic disorder that mostly affects boys because the gene is on the X chromosome. The gene here makes a transporter protein called MCT8, which carries thyroid hormone into cells. When that transporter...

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New Rare Disease Center of Excellence and FDA Advances in Rare Disease

September 29, 2026 • 9 mins

This week on The CheckRare Brief, we are joined by Eugene Lee, Head of CMI Media Group’s Rare Disease Division, for an industry perspective on some of the biggest developments in rare disease.

CMI Media Group recently launched a Specialized Rare Disease Center of Excellence to scale precision marketing to smaller pharmaceutical companies. CMI is well known for its media and communications capabilities in the heal...

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FDA Approves New Therapies for MPS IIIA and SMA

September 23, 2026 • 8 mins

The FDA announced the approval of Isembyld (apitegromab-mstn) to treat patients 2 yrs and older with spinal muscular atrophy who are currently receiving SMN2-targeted therapy.

SMA is a genetic condition in which patients lose spinal muscle function due to a mutation in the SMN1 gene. There are three therapies for this condition: one is a gene therapy that replaces the SMN1 gene, and the other two, significantly cheaper thera...

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First-Line Therapy Approved for Rare Lung Cancer

September 15, 2026 • 8 mins

This week, three developments highlight the rapidly changing rare disease treatment landscape: a Phase 3 setback in Angelman syndrome, continued competition in hereditary angioedema, and an FDA decision expanding access to a targeted lung cancer therapy.

Angelman Syndrome: Phase 3 Trial Misses Its Marks
Ultragenyx announced results from its Phase 3 Aspire study of apazunersen in Angelman syndrome. The trial did not meet i...

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FDA Approves Rare Disease Treatments as Huntington’s Gene Therapy Advances

September 9, 2026 • 9 mins

In this episode of The CheckRare Brief, we discuss two recent FDA approvals for rare diseases, including Zanvastro for Alexander disease and Besremi for essential thrombocythemia (ET). We also examine a new gene therapy application for Huntington’s disease and what it could mean for patients with this devastating inherited disorder.

The FDA has approved Zanvastro, the first drug indicated to treat patients with Alexand...

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New FDA Approvals for Rare Diseases: From Autoimmune Disorders to Pancreatic Cancer

September 3, 2026 • 9 mins

In this episode of The CheckRare Brief, we discuss four recent FDA approvals, including LISRAYA for dermatomyositis and Imaavy for warm autoimmune hemolytic anemia (wAIHA), Mimrylo for polycythemia vera (PV), and Rasonque for pancreatic cancer. We also examine the recent measles outbreak in Pennsylvania.

The FDA has approved Lisraya and Imaavy to treat two different autoimmune diseases – dermatomyositis and wAIHA. Hist...

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Getting a Rare Disease Drug Approved Is Only Half the Battle | The CheckRare Brief Ep

August 26, 2026 • 8 mins

FDA approval is a major milestone, but for patients with rare diseases, it is only the beginning. In this episode of The CheckRare Brief, we look at two new rare disease approvals, ongoing drug supply problems affecting patients, and a major acquisition that illustrates how rare disease therapies move from small biotech companies into the hands of larger pharmaceutical companies.

The US Food and Drug Administration (FDA) gra...

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FDA Approves Multiple Myeloma Drug; FDA Denies Neuroendocrine Drug; Safety Concerns For Prader-Willi Drug

August 19, 2026 • 8 mins

On this week’s episode of The CheckRare Brief, we discuss FDA’s approval of Zenbexus (iberdomide) to treat patients with multiple myeloma, the complete response letter issued to ITM-11 to treat patients with neuroendocrine tumors, and safety concerns about VYKAT XR for Prader-Willi syndrome (PWS).
The US Food and Drug Administration (FDA) granted accelerated approval to Bristol Myers Squibb’s iberdomide to trea...

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FDA Approves New Narcolepsy Treatment

August 12, 2026 • 7 mins

On this week’s episode of The CheckRare Brief, we discuss FDA’s approval of Orzeyful (oveporexton) to treat patients with narcolepsy type 1, CAMP4’s first-in-human clinical trial for patients with SYNGAP1-related disorders, and BioMarin’s termination for their ENPP1 deficiency program following mixed results from their phase 3 clinical trial.

The US Food and Drug Administration (FDA) approved Takeda&r...

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Episode 1: FDA Advisory Board’s Concerns About New Duchenne Drug, Brain Fog in Immune Thrombocytopenia; August is SMA Awareness Month

August 5, 2026 • 7 mins

The CheckRare Brief delivers a concise summary of the week's most important developments in rare diseases. Each episode covers FDA approvals, clinical trial updates, conference highlights, scientific publications, and industry news that matter to healthcare professionals, researchers, advocates, and the rare disease community.

Madaline Spencer, Podcast Producer; James Radke, PhD; Education Director; Peter Ciszewski, Fo...

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The CheckRare Brief

Episodes

.css-14f5ked{margin:0;word-break:break-word;display:-webkit-box;-webkit-box-orient:vertical;box-orient:vertical;-webkit-line-clamp:2;overflow:hidden;}Ultragenyx Sells PRV for $210M | The CheckRare Brief Ep 11

.css-r6mb8g{margin:0;word-break:break-word;display:-webkit-box;-webkit-box-orient:vertical;box-orient:vertical;-webkit-line-clamp:1;overflow:hidden;}Therapies for MCT8 Deficiency and Nephrotic Syndrome Get FDA Approval

New Rare Disease Center of Excellence and FDA Advances in Rare Disease

FDA Approves New Therapies for MPS IIIA and SMA

First-Line Therapy Approved for Rare Lung Cancer

FDA Approves Rare Disease Treatments as Huntington’s Gene Therapy Advances

New FDA Approvals for Rare Diseases: From Autoimmune Disorders to Pancreatic Cancer

Getting a Rare Disease Drug Approved Is Only Half the Battle | The CheckRare Brief Ep

FDA Approves Multiple Myeloma Drug; FDA Denies Neuroendocrine Drug; Safety Concerns For Prader-Willi Drug

FDA Approves New Narcolepsy Treatment

Episode 1: FDA Advisory Board’s Concerns About New Duchenne Drug, Brain Fog in Immune Thrombocytopenia; August is SMA Awareness Month

Popular Podcasts

Ultragenyx Sells PRV for $210M | The CheckRare Brief Ep 11

Therapies for MCT8 Deficiency and Nephrotic Syndrome Get FDA Approval