August 2023 – Page 2 – Patient Worthy

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Sudden Arrhythmia Death Syndromes Foundation (SADS) Educates FDA on Living with ARVC/D Through Externally-Led Patient-Focused Drug Development Meeting

Post author:Patient Worthy Contributor
Post published:August 24, 2023
Post category:Arrhythmogenic right ventricular dysplasia Sudden Arrhythmia Death Syndrome

This press release was provided by the Sudden Arrhythmia Death Syndrome Foundation (SADS), a Patient Worthy partner organization. Key facts: ● Externally-led patient-focused drug development meeting (EL-PFDD) with FDA hosted…

Chronic osteomyelitis in the clavicle. Sourced from: Shannonpercivalsmith / CC BY (https://creativecommons.org/licenses/by/3.0)

OI Has Led to More Than 82 Broken Bones for This 6-Year-Old

Post author:Jessica Lynn
Post published:August 24, 2023
Post category:Osteogenesis Imperfecta

For many people, a broken bone would severely interrupt their life. But six-year-old Avery Balcazar has learned to live with it—even when it’s tough. As reported by CBC News, Balcazar…

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Doctor Writes Book About Personal Experience with Lupus

Post author:Jessica Lynn
Post published:August 24, 2023
Post category:Lupus

When Dr. Niranjana Parthasarathi, M.D., was in medical school at Virginia Commonwealth University (VCU), she still remembers a lesson that changed her life. Her course introduced her to systemic lupus…

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Paralyzed with Pompe Disease, He Took Control, Gained Strength, and Increased Muscle

Post author:Rose Duesterwald
Post published:August 23, 2023
Post category:Pompe Disease

It was the second time 26-year-old Bruce Campbell had to deal with paralysis. At the age of six, he was paralyzed by Guillian-Barre syndrome which caused an attack on…

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Hybrid Work and the Sandwich Generation of Caregivers

Post author:Rose Duesterwald
Post published:August 23, 2023
Post category:Rare Disease

Sounds like ‘food for thought’. The term ‘sandwich generation’ describes family members who are usually between the ages of 45 and 55 and are responsible for raising their children…

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ICYMI: TAK-994 Reduced Sleepiness and Cataplexy in Narcolepsy – But Is It Safe?

Post author:Jessica Lynn
Post published:August 23, 2023
Post category:Narcolepsy

A Phase 2 clinical study found TAK-994 to be relatively effective in reducing symptoms like excessive daytime sleepiness (EDS) and cataplexy (sudden, uncontrollable muscle weakness often triggered by strong…

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Rare Community Profiles: Fiercely Advocating for the SMA Community: Why Amanda Will Never Stop Fighting for her Son Kayden

Post author:Jessica Lynn
Post published:August 23, 2023
Post category:Spinal Muscular Atrophy

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

Photo courtesy of IndoUSRare

Global Registries Could Change the Lives of Hundreds of Millions of Rare Disease Patients Everywhere

Post author:Patient Worthy Contributor
Post published:August 22, 2023
Post category:Rare Disease

Authored by Dr. Harsha Rajasimha, Founder and Executive Chairman, IndoUSrare. Co-Authored by Dr. Padma Rammoorthy, Medical Consultant, IndoUSrare. The diagnostic journey for patients with rare diseases often becomes a prolonged odyssey,…

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MAb-AR20.5 Earns Orphan Drug Designation for Pancreatic Cancer

Post author:Jessica Lynn
Post published:August 22, 2023
Post category:Pancreatic Cancer

Despite advances in research and the advent of multiple investigational treatments, pancreatic cancer is still incredibly difficult to treat. The five-year survival rate sits at just 12%. Existing therapies…

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Actress Francia Raisa Shares Her PCOS Experience

Post author:Jessica Lynn
Post published:August 22, 2023
Post category:Polycystic Kidney Disease

Actress Francia Raisa is well-known for her roles in Grown-Ish, Bring It On: All Or Nothing, The Secret Life of the American Teenager, and How I Met Your Father. She’s…

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In Her 9th Osteosarcoma Relapse, This Young Woman Continues to Fight

Post author:Jessica Lynn
Post published:August 22, 2023
Post category:Osteosarcoma

When Camille Wahl was a child, she loved Irish step dance. She translated her love physically, becoming a competitive dancer until she was ten years old. It was then, reports…

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Do Cross-Over Arms in Clinical Oncology Trials Benefit Patients?

Post author:James Moore
Post published:August 21, 2023
Post category:Cholangiocarcinoma

The Cholangiocarcinoma Foundation has recently released a white paper titled “Cross-over arms in oncology clinical trials: An important consideration for patients.” This paper highlights a non-traditional clinical trial approach that…

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CB-010 Shows Promise for B-Cell Non-Hodgkin Lymphoma, Study Shows

Post author:Jessica Lynn
Post published:August 21, 2023
Post category:Non-Hodgkin lymphoma Non-Hodgkin's Lymphoma

Recent results are available from the dose escalation portion of the Phase 1 ANTLER study. In reporting from Targeted Oncology, Jordyn Sava shares that the study included 16 participants living…

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Selinexor Granted Fast Track Designation for Myelofibrosis

Post author:Jessica Lynn
Post published:August 21, 2023
Post category:Myelofibrosis primary myelofibrosis Timely

In the United States, the FDA grants Fast Track designation to drugs that treat rare and serious conditions or fill unmet needs. The designation helps to facilitate and expedite the…

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Cubs Fan with PKD Goes to Wrigley Field in Search of Kidney Donor

Post author:Jessica Lynn
Post published:August 21, 2023
Post category:Polycystic Kidney Disease

When Christine Todd learned that her kidneys were failing, she was frightened and concerned. Todd has polycystic kidney disease (PKD), an inherited condition characterized by cysts that develop in the…

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An Increase in the Development of Treatments for Rare Diseases

Post author:Rose Duesterwald
Post published:August 20, 2023
Post category:Rare Disease

The number of novel drugs approved by the FDA has doubled in the ten-year period from 2012 through 2020. Investment in investigational drugs for rare diseases appears to be continuing…

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A Novel Formulation of Vyjuvek Helped One Teen with DEB Reclaim His Eyesight

Post author:Jessica Lynn
Post published:August 20, 2023
Post category:Dystrophic epidermolysis bullosa

In May 2023, the U.S. Food and Drug Administration (FDA) approved Vyjuvek, a gene therapy, for dystrophic epidermolysis bullosa (DEB). This treatment has brought support to numerous people living with…

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Researchers Dive Deeper into Understanding the Ultra-Rare XLP-2

Post author:Jessica Lynn
Post published:August 19, 2023
Post category:X-linked lymphoproliferative syndrome

As our understanding of genetics increases, so does our awareness of the multitude of genetic diseases—many of which continue to be discovered to this day. While X-linked lymphoproliferative disease (XLP)…

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Nemolizumab Reduces Pruritus (Itchiness) in Children with Atopic Dermatitis

Post author:Jessica Lynn
Post published:August 19, 2023
Post category:Atopic dermatitis

An estimated 25% of children globally are affected by atopic dermatitis; you may known this skin condition by its more colloquial name, "eczema." Often manifesting by five years old, atopic…

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Mavorixafor is Safe and Effective for Treating WHIM Syndrome, Trial Data Suggests

Post author:Jessica Lynn
Post published:August 18, 2023
Post category:WHIM syndrome

In the past, treatment for WHIM syndrome has focused on symptom management: treating infections and myelokathexis with antibiotics or granulocyte colony stimulating factor. Infusion can treat hypogammaglobulinemia and surgery…

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Gender Bias is Impacting Women with Hemophilia: How to Bridge the Gap

Post author:James Moore
Post published:August 18, 2023
Post category:Hemophilia

According to a story from Reuters Events, hemophilia treatment has seen significant advancements in recent years. However, female patients continue to face difficulties that often lead to disparities in quality…

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Phase II Narcolepsy Clinical Trial Halted After Severe Adverse Effects

Post author:James Moore
Post published:August 18, 2023
Post category:Narcolepsy

According to a story from Clinical Trials Arena, the pharmaceutical company Takeda recently released findings from its phase II clinical trial. This trial was evaluating the company's investigational drug TAK-994…

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Rare Classroom: Arginase Deficiency Disorder

Post author:James Moore
Post published:August 18, 2023
Post category:Arginase deficiency

Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…

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Swimmer with Osteogenesis Imperfecta Wins Gold & Silver at the Hartford Nationals

Post author:Jessica Lynn
Post published:August 17, 2023
Post category:Osteogenesis Imperfecta

If you’re looking for drive and determination, look no further. 14-year-old Sophia Nohre is ready to tackle any obstacle in her path—and she does so relentlessly. Her swimming coach, Adam…

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Positive Results in Phase 3 Chronic Inflammatory Demyelinating Polyneuropathy Trial

Post author:James Moore
Post published:August 17, 2023
Post category:Chronic Inflammatory Demyelinating Polyneuropathy

According to a story from Business Wire, the pharmaceutical company Takeda recently presented results from its phase 3 clinical trial evaluating HYQVIA (Human Immune Globulin Infusion 10% with Recombinant Human…

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Sudden Arrhythmia Death Syndromes Foundation (SADS) Educates FDA on Living with ARVC/D Through Externally-Led Patient-Focused Drug Development Meeting

OI Has Led to More Than 82 Broken Bones for This 6-Year-Old

Doctor Writes Book About Personal Experience with Lupus

Paralyzed with Pompe Disease, He Took Control, Gained Strength, and Increased Muscle

Hybrid Work and the Sandwich Generation of Caregivers

ICYMI: TAK-994 Reduced Sleepiness and Cataplexy in Narcolepsy – But Is It Safe?

Rare Community Profiles: Fiercely Advocating for the SMA Community: Why Amanda Will Never Stop Fighting for her Son Kayden

Global Registries Could Change the Lives of Hundreds of Millions of Rare Disease Patients Everywhere

MAb-AR20.5 Earns Orphan Drug Designation for Pancreatic Cancer

Actress Francia Raisa Shares Her PCOS Experience

In Her 9th Osteosarcoma Relapse, This Young Woman Continues to Fight

Do Cross-Over Arms in Clinical Oncology Trials Benefit Patients?

CB-010 Shows Promise for B-Cell Non-Hodgkin Lymphoma, Study Shows

Selinexor Granted Fast Track Designation for Myelofibrosis

Cubs Fan with PKD Goes to Wrigley Field in Search of Kidney Donor

An Increase in the Development of Treatments for Rare Diseases

A Novel Formulation of Vyjuvek Helped One Teen with DEB Reclaim His Eyesight

Researchers Dive Deeper into Understanding the Ultra-Rare XLP-2

Nemolizumab Reduces Pruritus (Itchiness) in Children with Atopic Dermatitis

Mavorixafor is Safe and Effective for Treating WHIM Syndrome, Trial Data Suggests

Gender Bias is Impacting Women with Hemophilia: How to Bridge the Gap

Phase II Narcolepsy Clinical Trial Halted After Severe Adverse Effects

Rare Classroom: Arginase Deficiency Disorder

Swimmer with Osteogenesis Imperfecta Wins Gold & Silver at the Hartford Nationals

Positive Results in Phase 3 Chronic Inflammatory Demyelinating Polyneuropathy Trial

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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