rare disease – Page 30 – Patient Worthy

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Avoid These 5 Real-World Data Pitfalls When Planning your Patient Registry, Natural History, or Other Clinical Study for a Rare Disease

Post author:James Moore
Post published:December 16, 2020
Post category:Rare Disease

Author: Harsha K Rajasimha, MS, Ph.D. Editor: Sharlene Brown, Ph.D. When clinical investigators and study coordinators start planning a new study design, speed and efficiency are paramount. Running a clinical…

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Pharma & Patient USA 2020: Strengthening Connections Between Patients and Medicine During COVID-19

Post author:James Moore
Post published:December 9, 2020
Post category:Rare Disease

From December 1-4, 2020, Reuters hosted a virtual conference called Pharma & Patient USA 2020. The primary focus of this event was furthering patient centricity in disease development as well…

Unanimously Passed Bill Improves the Orphan Drug Act

Post author:Trudy Horsting
Post published:December 3, 2020
Post category:Rare Disease

The Orphan Drug Act originated in 1983 as an incentive for pharmaceutical companies who worked on developing treatments for rare disease. Since rare diseases are well, rare, choosing to work…

Cambridge Rare Disease Network’s RAREfest20

Post author:James Moore
Post published:November 28, 2020
Post category:

The Cambridge Rare Disease Network presents RARE Fest 2020 Engage.Educate.Empower. November 28, 2020 In light of the COVID-19 pandemic, RAREfest20 will be celebrated as a live virtual festival that will…

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RNA Interference and the Treatment of Rare Disease

Post author:James Moore
Post published:November 27, 2020
Post category:hepatic porphyria Hereditary ATTR Amyloidosis Rare Disease

On November 19, 2020, Research!America hosted an informational webinar titled "From Nobel Winning Science to Next Generation Treatment: Tracing the Path of a Rare Disease Breakthrough using RNAi." This webinar focused…

5 Questions I Ask Myself at Thanksgiving with Rare Disease

Post author:Patient Worthy Contributor
Post published:November 26, 2020
Post category:Dysautonomia Lyme Disease POTS

Thanksgiving is a time of gratitude, family and maybe a little gluttony. It was always my favorite time of the year in high school and college. Now, while I still…

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The Autoimmune Registry Releases Comprehensive List of Autoimmune Diseases

Post author:James Moore
Post published:November 25, 2020
Post category:Multiple Sclerosis Myasthenia Gravis Vasculitis

According to a story from PR Newswire, The Autoimmune Registry, Inc. (ARI) has recently released its first ever autoimmune diseases list. This list includes a total of over 150 distinct…

Navigating Rare Disease Clinical Trials in a Global Pandemic Means Putting Patients First

Post author:Trudy Horsting
Post published:November 24, 2020
Post category:Rare Disease

Navigating a Rare Disease in a Pandemic The COVID-19 pandemic has affected everyone, but those already living with a rare disease have unique challenges. With a compromised immune system, they…

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Consider Rare Disease Families at Holiday Time

Post author:Denise Crompton
Post published:November 23, 2020
Post category:MPS III (Sanfilippo Syndrome)Mucolipidosis Type III Rare Disease

We often hear people talking about the stress they are feeling during the holidays, while those in the rare disease community silently think, "You have no idea!" During this season,…

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FDA Approves of Status Epilepticus Treatment, SESQUIENT

Post author:Kendall Mason
Post published:November 18, 2020
Post category:Status epilepticus

Sedor Pharmaceuticals, a partner of Ligand Pharmaceuticals, has recently received FDA approval for SESQUIENT, its status epilepticus treatment. It has been cleared for the treatment of both adult and pediatric…

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Rare Classroom: POMC Deficiency

Post author:James Moore
Post published:November 18, 2020
Post category:POMC deficiency Rare Disease

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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Hope for a New IgA Nephropathy Treatment Following Phase III Study

Post author:James Moore
Post published:November 18, 2020
Post category:IgA Nephropathy Proteinuria

According to a story from pharmalive.com, the drug company Calliditas Therapeutics will plan to seek approval for its therapy Nefecon from the US Food and Drug Administration (FDA). This approval…

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New Combination for HRD-Positive Ovarian Cancer Gets EU Approval

Post author:James Moore
Post published:November 18, 2020
Post category:Ovarian Cancer

According to a story from Cancer Network, the European Commission has recently approved a two-part combination treatment for advanced, homologous recombination deficiency (HRD)-positive ovarian cancer. This first-line maintenance treatment consists…

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CAR-T Therapy for Multiple Myeloma Trial Marred by Patient Death

Post author:Kendall Mason
Post published:November 17, 2020
Post category:Multiple Myeloma

According to Biospace, Allogene's trial of ALLO-715 for relapsed/refractory multiple myeloma was marred by a patient death. Data from this trial is slated to be presented in December at the…

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Emergency Planning For People With HAE and AAE

Post author:Patient Worthy Contributor
Post published:November 17, 2020
Post category:Hereditary Angiodema

People with hereditary and acquired angioedema have unpredictable attacks of swelling/inflammation/pain, most of which can be managed with rescue meds like Firazyr, Berinert, Kalbitor, and Rhucin, plus a prescribed med…

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CCA Gives Tips for Transitioning into Adulthood with a Craniofacial Difference

Post author:Trudy Horsting
Post published:November 17, 2020
Post category:Cranioacrofacial syndrome Rare Disease

Children's Craniofacial Association (CCA) has posted tips for transitioning to adulthood for those with craniofacial differences. We've summarized some of their recommendations, but you can read the full article here.…

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This Drug Could Boost Duchenne Muscular Dystrophy Treatment

Post author:James Moore
Post published:November 17, 2020
Post category:Duchenne Muscular Dystrophy Muscular Dystrophy

According to a story from Newswise, trial results released in the journal PLOS Medicine suggest that the drug vamorolone could provide an enhancement in the effectiveness of corticosteroid therapy in patients…

CymaBay Therapeutics Announces Results of Global Phase 3 ENHANCE Study for Primary Biliary Cholangitis

Post author:Rose Duesterwald
Post published:November 17, 2020
Post category:Primary Biliary Cholangitis Rare Disease

CymaBay Therapeutics, a biopharmaceutical company based in Newark, CA, develops and provides access to novel therapies with a recent focus on primary biliary cholangitis (PBC). The company is currently developing…

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AMO-02, a Congenital Myotonic Dystrophy Treatment, Granted Rare Pediatric Disease Designation

Post author:Kendall Mason
Post published:November 17, 2020
Post category:Myotonic Dystrophy

The FDA has recently granted the Rare Pediatric Disease (RPD) designation to AMO-02, a treatment for congenital myotonic dystrophy. As there is currently an unmet medical need for this condition,…

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Year Two of This Alport Syndrome Trial Concludes with Encouraging Findings

Post author:James Moore
Post published:November 16, 2020
Post category:Alport syndrome Autosomal Dominant Polycystic Kidney Disease Chronic Kidney Disease IgA Nephropathy

According to a story from GlobeNewswire, the biopharmaceutical company Reata Pharmaceuticals, Inc., recently announced that year two of its phase 3 clinical trial has been completed. This trial is testing…

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Two New Clinical Trials for Vosoritide, an Achondroplasia Treatment

Post author:Kendall Mason
Post published:November 16, 2020
Post category:Achondroplasia

BioMarin plans to expand its clinical program for vosoritide, its treatment for achondroplasia. This expansion includes two new phase 2 trials, the first of which will evaluate vosoritide in infants…

In Mozambique A Mural Writes, “People With Albinism are the Same as You!”

Post author:Sunniva Bean
Post published:November 16, 2020
Post category:Albinism Rare Disease

As reported by the Human Rights Watch; in Mozambique, a grassroots organization to support people with albinism is using murals to spread awareness and a positive image of the disease.…

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Study Identifies Clinical Characteristics of Chronic Nonbacterial Osteomyelitis

Post author:James Moore
Post published:November 16, 2020
Post category:Chronic recurrent multifocal osteomyelitis Osteomyelitis

According to a story from EurekAlert! a recent study presented at the annual meeting of the American College of Rheumatology describes some of the key clinical feature of chronic nonbacterial…

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Rare Classroom: Maroteaux-Lamy Syndrome

Post author:James Moore
Post published:November 16, 2020
Post category:MPS VI MPS VI (Maroteaux-Lamy syndrome)Mucopolysaccharidosis Rare Disease

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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A Combative Combination: Fighting Osteomyelitis with Stem Cells and Antibiotics

Post author:Patient Worthy Contributor
Post published:November 13, 2020
Post category:Osteomyelitis Rare Disease

by Danielle Bradshaw from In The Cloud Copy Bone grafts, although very helpful, can result in hard to treat infections and typically result in the patient needing to be put…

Avoid These 5 Real-World Data Pitfalls When Planning your Patient Registry, Natural History, or Other Clinical Study for a Rare Disease

Pharma & Patient USA 2020: Strengthening Connections Between Patients and Medicine During COVID-19

Unanimously Passed Bill Improves the Orphan Drug Act

Cambridge Rare Disease Network’s RAREfest20

RNA Interference and the Treatment of Rare Disease

5 Questions I Ask Myself at Thanksgiving with Rare Disease

The Autoimmune Registry Releases Comprehensive List of Autoimmune Diseases

Navigating Rare Disease Clinical Trials in a Global Pandemic Means Putting Patients First

Consider Rare Disease Families at Holiday Time

FDA Approves of Status Epilepticus Treatment, SESQUIENT

Rare Classroom: POMC Deficiency

Hope for a New IgA Nephropathy Treatment Following Phase III Study

New Combination for HRD-Positive Ovarian Cancer Gets EU Approval

CAR-T Therapy for Multiple Myeloma Trial Marred by Patient Death

Emergency Planning For People With HAE and AAE

CCA Gives Tips for Transitioning into Adulthood with a Craniofacial Difference

This Drug Could Boost Duchenne Muscular Dystrophy Treatment

CymaBay Therapeutics Announces Results of Global Phase 3 ENHANCE Study for Primary Biliary Cholangitis

AMO-02, a Congenital Myotonic Dystrophy Treatment, Granted Rare Pediatric Disease Designation

Year Two of This Alport Syndrome Trial Concludes with Encouraging Findings

Two New Clinical Trials for Vosoritide, an Achondroplasia Treatment

In Mozambique A Mural Writes, “People With Albinism are the Same as You!”

Study Identifies Clinical Characteristics of Chronic Nonbacterial Osteomyelitis

Rare Classroom: Maroteaux-Lamy Syndrome

A Combative Combination: Fighting Osteomyelitis with Stem Cells and Antibiotics

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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