Clinical Insights
Making Travel Accessible: SMAshing My Limits Partners With Becoming rentABLE
Tracey Dawson, PhD, Senior Vice President, US Therapeutic Area Head Neuroscience at Novartis, discusses the collaboration between Novartis, SMAshing My Limits, and Becoming rentABLE, striving to make travel accessible. Spinal muscular atrophy (SMA) is a...
Getting a Rare Disease Drug Approved Is Only Half the Battle | The CheckRare Brief Ep 4
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.
FDA Approves First Treatment for Warm Autoimmune Hemolytic Anemia (wAIHA)
The US Food and Drug Administration (FDA) has approved Imaavy (nipocalimab) for the treatment of patients ages 12 years and older with warm autoimmune hemolytic anemia (wAIHA) who are currently or previously treated with corticosteroids.
Addressing Patient Experiences With IgA Nephropathy
Gaia Coppock, MD, Nephrologist and Associate Professor of Clinical Medicine at the University of Pennsylvania, discusses patient experiences with IgA nephropathy (IgAN).
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FDA Approves Pasatru (Garetosmab) for Adults With Fibrodysplasia Ossificans Progressiva
The US Food and Drug Administration (FDA) has approved Pasatru (garetosmab-grts) to reduce formation of new heterotopic ossification (HO) lesions and clinician-assessed flare-ups in adults with...
FDA Grants Accelerated Approval to AAV8-Based Gene Therapy (Genglycos) for Patients With Glycogen Storage Disease Type 1a
The US Food and Drug Administration (FDA) has granted accelerated approval for Genglycos (pariglasgene brecaparvovec-opnr), also known as DTX401, in patients eight years and older with glycogen...
Patient Perspective: Diagnostic Journey With IgA Nephropathy
Byron Wade, a patient with IgA nephropathy (IgAN), discusses his personal diagnostic journey and a recent study looking at other patients’ experiences. IgAN is characterized by the...
FDA Approves Multiple Myeloma Treatment, Denies Neuroendocrine Tumors Treatment
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...
Importance of Patient Listening in The Development of Rare Disease Treatments
Matt Trudeau, President of ITF Therapeutics, discusses the importance of patient listening in the development of Duvyzat (givinostat) for Duchenne muscular dystrophy (DMD). DMD is a...
Rare Endocrine Exchange: Why This Conversation Matters
Rare endocrine disorders can be difficult to recognize, challenging to diagnose, and even harder for patients to navigate. For many patients, the journey to an accurate diagnosis can take years....
FDA Approves Iberdomide Combination Therapy for Relapsed/Refractory Multiple Myeloma
The US Food and Drug Administration (FDA) has granted accelerated approval to iberdomide (Zenbexus) in combination with daratumumab and hyaluronidase-fihj and dexamethasone for adults with multiple...
Topline Results from the MonumenTAL-6 Clinical Trial in Adults With Relapsed/Refractory Multiple Myeloma
Ajay K. Nooka, MD, MPH, FACP, Director of the Myeloma Program at the Emory University School of Medicine, discusses topline results from the MonumenTAL-6 clinical trial testing Tecvayli...
Follow-Up Data of Safusidenib in Patients With IDH-Mutant Gliomas
Alexandra Miller, MD, PhD, Chief of Neuro-Oncology and Co-Director of the Brain and Spine Tumor Center at the Perlmutter Cancer Center at NYU Langone Health, discusses follow-up data on the safety...
FDA Approves New Narcolepsy Treatment | The CheckRare Brief
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...
ATLAS-OLE Subgroup Analysis of Fitusiran in Patients With Hemophilia
Guy Young, MD, Director of the Hemostasis and Thrombosis Program at Children's Hospital Los Angeles, discusses an ATLAS-OLE subgroup analysis of fitusiran in patients with hemophilia. ...
Safety and Efficacy of Kygevvi in Thymidine Kinase 2 Deficiency
Caterina Garone, PhD, MD, Associate Professor at the University of Bologna, discusses safety and efficacy data on Kygevvi (doxecitine and doxribtimine) in patients with thymidine kinase 2 deficiency...
Now Enrolling: GALILEO-3 Trial Evaluating FLT201 for Adults With Gaucher Disease Type 1
Priya Kishnani, MD, Professor and Chief of the Division of Medical Genetics at Duke University, discusses the GALILEO-3 clinical trial of FLT201 in adults with Gaucher disease type 1 (GD1). ...
FDA Advisory Board’s Concerns About New Duchenne Drug, Brain Fog in Immune Thrombocytopenia
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,...
FDA Approves Orzeyful (Oveporexton) for the Treatment of Adults With Narcolepsy Type 1
The US Food and Drug Administration (FDA) has approved Orzeyful (oveporexton) for the treatment of adults with narcolepsy type 1 Narcolepsy is a chronic neurological disorder characterized by an...
LOTUS Study in Patients With Rett Syndrome Treated With Daybue (Trofinetide)
Ryan Bucco, PharmD, Vice President of Medical Affairs in Rare Disease at Acadia, discusses the LOTUS study in patients with Rett syndrome treated with Daybue (trofinetide). Rett syndrome is a...
Results From the ENERGY Clinical Trial Testing Imaavy (Nipocalimab) in Warm Autoimmune Hemolytic Anemia (wAIHA)
Bruno Fattizzo, MD, Department of Oncology and Hemato-Oncology at the University of Milan, discusses results from the ENERGY clinical trial of Imaavy (nipocalimab) in warm autoimmune hemolytic...
Initiation of Phase 3 FORZETTO Clinical Trial Testing Zeleciment Rostudirsen in Duchenne Muscular Dystrophy
Doug Kerr, MD, PhD, MBA, Chief Medical Officer of Dyne Therapeutics, discusses the initiation of the phase 3 FORZETTO trial of zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251), in...
August Is SMA Awareness Month
August is SMA Awareness Month, a time dedicated to increasing understanding of spinal muscular atrophy (SMA) and supporting the individuals and families impacted by this rare genetic neuromuscular...
Phase 2a GATEWAY Trial of PORT-77 in Patients With Erythropoietic Protoporphyria Shows Promise
Pete Schmidt, MD, MSc, Chief Medical Officer of Portal Therapeutics, discusses data from the phase 2a GATEWAY trial testingPORT-77, an ABCG2 inhibitor, in patients with erythropoietic protoporphyria...
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Rare Endocrine Exchange: Diagnosing Rare Endocrine Disorders
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Making Travel Accessible: SMAshing My Limits Partners With Becoming rentABLE
Robert Rapaport, MD, discusses severe primary IGF-1 deficiency.
Learn the differences between this rare disease and growth hormone (GH) deficiency in patients with short stature at https://checkrare.com/primary-igf-1-deficiency-causes-early-detection-and-treatment/
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Predicting Treatment Response Outcomes With Proteomic and Machine Learning Analyses in MG
Kleefstra Syndrome: Development of RNA Amplifier Therapy
Robert Rapaport, MD, discusses severe primary IGF-1 deficiency.
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#CheckRare ...#IGF1Defiency #SIGF1D #RareGenetic #RareEndocrine
Getting a Rare Disease Drug Approved Is Only Half the Battle | The CheckRare Brief Ep 4
In the first episode of the Rare Endocrine Exchange, Margarita Ochoa-Maya, MD, discusses when further diagnostic testing should be done. Learn more about our new podcast and why this conversation matters at https://checkrare.com/multimedia/rare-endocrine-exchange/or wherever you get your ...podcasts.
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In the first episode of the Rare Endocrine Exchange, Margarita Ochoa-Maya, MD, discusses when further diagnostic testing should be done. Learn more about our new podcast and why this conversation matters at the link in our bio or wherever you get your podcasts.
#CheckRare ...#RareEndocrineExchange #RareEndocrine #RareEndocrineDisorders #Endocrine #Podcast
Rare Endocrine Exchange: Diagnosing Rare Endocrine Disorders
FDA Approves Multiple Myeloma Drug; FDA Denies Neuroendocrine Drug; Safety Concerns For Prader-W...
Listen to episode 3 of The CheckRare Brief, available now wherever you get your podcasts!
Link in our bio.
#CheckRare #TheCheckRareBrief #RareDiseaseNews
Listen to episode 3 of The CheckRare Brief, available now wherever you get your podcasts!
https://checkrare.com/fda-approves-multiple-myeloma-treatment-denies-neuroendocrine-tumors-treatment/
#CheckRare #TheCheckRareBrief #RareDiseaseNews
Rare Endocrine Exchange: Why This Conversation Matters
FDA Approves Multiple Myeloma Drug; FDA Denies Neuroendocrine Drug; Safety Concerns For Prader-W...












Making Travel Accessible: SMAshing My Limits Partners With Becoming rentABLE
CheckRare 15 hours ago