June 2024 – Patient Worthy

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Pfizer Acknowledges the Death of a Young Boy Who Died One Year After Participating in its Phase II Clinical Trial to Treat DMD

Post author:Rose Duesterwald
Post published:June 28, 2024
Post category:Duchenne Muscular Dystrophy

Pfizer has recently paused its Phase III clinical trial that has been assessing the Duchenne muscular dystrophy candidate fordadistrogene movaparvovec. The Phase II DAYLIGHT trial (NCT05429372), enrolled ten boys ages…

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FDA Approves Dosage Boost for Lambert-Eaton Myasthenic Syndrome Drug FIRDAPSE

Post author:James Moore
Post published:June 28, 2024
Post category:Lambert-Eaton Myasthenic Syndrome

According to a story from Globe Newswire, the biopharmaceutical company Catalyst Pharmaceuticals has recently announced an update to the company's supplemental New Drug Application (sNDA) for its drug amifampridine (marketed…

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FDA Removes Clinical Hold on Nomlabofusp Study for Friedreich’s Ataxia

Post author:Jessica Lynn
Post published:June 28, 2024
Post category:Friedreich ataxia Friedreich's Ataxia Friedreich's ataxia

Three years after the U.S. Food and Drug Administration (FDA) first placed a full clinical hold on Larimar Therapeutics’ Phase 2 clinical study, the hold has officially been lifted. The…

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ICYMI: FDA Approves BKEMV, A Soliris Biosimilar, for aHUS and PNH

Post author:Jessica Lynn
Post published:June 26, 2024
Post category:Atypical hemolytic uremic syndrome paroxysmal nocturnal hemoglobinuria

Have you ever heard of a biosimilar? A biosimilar is essentially a biologic that is highly similar in structure and function to a reference product. Although this might seem technical, it really…

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The Impact of Artificial Intelligence: Predicting Visual Acuity in DME Following Vabysmo Treatment

Post author:Jessica Lynn
Post published:June 26, 2024
Post category:Diabetic macular edema

Artificial intelligence (AI) is changing the way we live. It seems rare to find an industry where AI has not made at least a preliminary impact. In politics, AI-generated cartoons…

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Science Simplified: CRISPR

Post author:Patient Worthy Contributor
Post published:June 26, 2024
Post category:Rare Disease SLC13A5 deficiency

Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science…

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Rare Fungal STI Trichophyton Mentagrophytes Type VII Has Reached the US

Post author:Jessica Lynn
Post published:June 25, 2024
Post category:Rare Disease

Trichophyton mentagrophytes type VII is a fungal species that are usually transmitted through environmental spores. This fungal infection normally causes skin issues in animals. But wait - there's more. Trichophyton…

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Making Better Choices When We Are in Pain and Suffering

Post author:Tom Seaman
Post published:June 25, 2024
Post category:Dystonia

If you have dystonia and chronic pain like I do, or one of many other thousands of health conditions, or just dealing with some tough life situations...you are not alone.…

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Rare Community Profiles: A New Treatment Option on the Horizon: Zevra Therapeutics’ Dan Gallo Discusses Arimoclomol, the NDA PDUFA Date, and the Needs of the NPC Community

Post author:Jessica Lynn
Post published:June 25, 2024
Post category:Niemann-Pick Disease Niemann-Pick Type C Disease

Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…

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Jenny Decker May Have Charcot-Marie-Tooth Disease, but She is Sailing Around the Globe

Post author:Rose Duesterwald
Post published:June 24, 2024
Post category:Charcot-Marie-Tooth disease

One year ago, Jenny Decker age 40 began her journey around the world sailing 3,200 miles to her first stop in Fiji. She reported to Practical Boat Owner that she…

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10,000 Mile Challenge: Uplifting Athletes Seeks to Support the Rare Disease Community

Post author:Jessica Lynn
Post published:June 24, 2024
Post category:Rare Disease

Get ready to lace up your sneakers, grab your swim goggles, hop on your bike, or move the way you move best - all for a great cause! Uplifting Athletes,…

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World Pneumothorax Day 2024: Spreading Rare Disease Awareness

Post author:James Moore
Post published:June 24, 2024
Post category:Birt-Hogg-DubÃ© syndrome (BHD)Ehlers-Danlos Syndrome Loeys-Dietz syndrome Lymphangioleiomyomatosis Marfan Syndrome Pneumothorax

June 24, 2024 is being recognized as World Pneumothorax Day, a time for spreading awareness about pneumothorax among the general public and in the medical field. Pneumothorax is a condition…

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Tardive Dyskinesia: Phase 3 Study Results Indicate Improvement with Use of INGREZZA®

Post author:Rose Duesterwald
Post published:June 21, 2024
Post category:Tardive Dyskinesia

This week Neurocrine Biosciences announced the results of its KINECT®-4 Phase 3 study of INGREZZA® capsules. Results of the trial were published in the Journal of Clinical Psychopharmacology. A long-term…

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Cartesian Therapeutics Earns Regenerative Medicine Advanced Therapy Designation for Myasthenia Gravis

Post author:Rose Duesterwald
Post published:June 21, 2024
Post category:Myasthenia Gravis

Cartesian Therapeutics, Inc., a biotechnology company, recently announced that the FDA has awarded the Regenerative Medicine Advanced Therapy (RMAT) designation to its lead product, Descartes-08, to treat myasthenia gravis (MG).…

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Healthy Skin Transplants Could Successfully Treat Epidermolytic Ichthyosis and Ichthyosis with Confetti

Post author:Jessica Lynn
Post published:June 21, 2024
Post category:Ichthyosis

You've heard of kidney transplants. You've heard of liver transplants. And you've probably even heard of lung transplants. But have you heard of skin transplants? In many cases, skin transplants…

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Singer Halsey Reveals Lupus and T-Cell Lymphoproliferative Disorder Diagnosis from 2 Years Ago

Post author:Jessica Lynn
Post published:June 20, 2024
Post category:Lupus

Rare diseases and chronic illnesses do not discriminate. They do not care if you are young or old, where you come from, or how much money you make. With an…

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FDA Approves Iqirvo for Primary Biliary Cholangitis (PBC)

Post author:Jessica Lynn
Post published:June 20, 2024
Post category:Primary Biliary Cholangitis

To the primary biliary cholangitis (PBC) community - it's time to rejoice! For the first time in nearly one decade, the U.S. Food and Drug administration (FDA) has approved a…

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2nd Annual Aces Fore Austin Golf Tournament Raises Rare Disease Funds, Awareness, and Hope

Post author:Jessica Lynn
Post published:June 20, 2024
Post category:Rare Disease

Parenthood is about raising the child you have, not the child you thought you'd have. Anthony and Lindsey Terling shared this message in their video "The Austin Way!" where the…

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Tafenoquine Granted Orphan Drug Designation for Acute Babesiosis

Post author:Jessica Lynn
Post published:June 18, 2024
Post category:Babesiosis

Tafenoquine is an anti-malarial drug that interferes with parasitic growth in the body. But could it be repurposed for use in another parasitic illness? That's what pharmaceutical company 60 Degrees…

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Indian Hospital Offers Free Spinal Muscular Atrophy (SMA) Testing

Post author:Jessica Lynn
Post published:June 18, 2024
Post category:Spinal Muscular Atrophy

Testing for rare conditions can be costly and time-consuming. In India, testing for spinal muscular atrophy (SMA), a rare genetic disorder, has traditionally cost between 3500 INR to 7000 INR…

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Rare Community Profiles: White Paper Discusses Access Barriers in IgG4-Related Disease (IgG4-RD)

Post author:Jessica Lynn
Post published:June 18, 2024
Post category:Rare Disease

Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…

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ICYMI: Neuraxpharm and Minoryx’s Special Information Film Raises Leukodystrophy Awareness

Post author:Jessica Lynn
Post published:June 17, 2024
Post category:Adrenoleukodystrophy Leukodystrophy

Rare Disease Day may be long past in February, but the fight for rare disease awareness continues. Earlier this year, specialty pharmaceutical company Neuraxpharm Group ("Neuraxpharm") and biotechnology company Minoryx…

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Physicians Identify Poor Disease Management in Patients with IBD

Post author:Jessica Lynn
Post published:June 17, 2024
Post category:Crohn's disease Inflammatory Bowel Disease ulcerative Colitis

Effective disease management in inflammatory bowel disease (IBD), which encompasses Crohn’s disease and ulcerative colitis, is incredibly important. Managing your condition helps you maintain a better quality-of-life, reduces uncomfortable or…

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Karen Overpowered Dystonia and Passes the Freedom, Like the Olympic Flame, to her Readers

Post author:Rose Duesterwald
Post published:June 17, 2024
Post category:Dystonia

Occasionally the acronym NEGU may appear in a social media post. It has been established as a rallying cry and support for people with cancer or other debilitating diseases. It…

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Can We Go Too Far with Self-Care?

Post author:Tom Seaman
Post published:June 14, 2024
Post category:Dystonia

If you have read my books or articles, you have probably noticed that I talk about self-care quite a bit. I think self-care is extremely important for anyone, whether they…

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Pfizer Acknowledges the Death of a Young Boy Who Died One Year After Participating in its Phase II Clinical Trial to Treat DMD

FDA Approves Dosage Boost for Lambert-Eaton Myasthenic Syndrome Drug FIRDAPSE

FDA Removes Clinical Hold on Nomlabofusp Study for Friedreich’s Ataxia

ICYMI: FDA Approves BKEMV, A Soliris Biosimilar, for aHUS and PNH

The Impact of Artificial Intelligence: Predicting Visual Acuity in DME Following Vabysmo Treatment

Science Simplified: CRISPR

Rare Fungal STI Trichophyton Mentagrophytes Type VII Has Reached the US

Making Better Choices When We Are in Pain and Suffering

Rare Community Profiles: A New Treatment Option on the Horizon: Zevra Therapeutics’ Dan Gallo Discusses Arimoclomol, the NDA PDUFA Date, and the Needs of the NPC Community

Jenny Decker May Have Charcot-Marie-Tooth Disease, but She is Sailing Around the Globe

10,000 Mile Challenge: Uplifting Athletes Seeks to Support the Rare Disease Community

World Pneumothorax Day 2024: Spreading Rare Disease Awareness

Tardive Dyskinesia: Phase 3 Study Results Indicate Improvement with Use of INGREZZA®

Cartesian Therapeutics Earns Regenerative Medicine Advanced Therapy Designation for Myasthenia Gravis

Healthy Skin Transplants Could Successfully Treat Epidermolytic Ichthyosis and Ichthyosis with Confetti

Singer Halsey Reveals Lupus and T-Cell Lymphoproliferative Disorder Diagnosis from 2 Years Ago

FDA Approves Iqirvo for Primary Biliary Cholangitis (PBC)

2nd Annual Aces Fore Austin Golf Tournament Raises Rare Disease Funds, Awareness, and Hope

Tafenoquine Granted Orphan Drug Designation for Acute Babesiosis

Indian Hospital Offers Free Spinal Muscular Atrophy (SMA) Testing

ICYMI: Neuraxpharm and Minoryx’s Special Information Film Raises Leukodystrophy Awareness

Physicians Identify Poor Disease Management in Patients with IBD

Karen Overpowered Dystonia and Passes the Freedom, Like the Olympic Flame, to her Readers

Can We Go Too Far with Self-Care?

Metastatic Breast Cancer: Navigating Grief

Rethinking What It Means to Live With Acromegaly

The Let’s Chat CAR T One-on-One Mentor Program: Speaking with Someone Who Understands What You Are Going Through

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