Updates

June 29, 2020June 29, 2020
CellCept, Cyclophosphamide Both Improve Patient-reported Outcomes in SSc-ILD, Phase 2 Data Show

CellCept (mycophenolate mofetil) and cyclophosphamide can both improve the health-related quality of life of people with systemic sclerosis-related interstitial lung disease, an analysis from ... Read more
June 23, 2020June 23, 2020
Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization ... Read more
June 22, 2020June 22, 2020
Rheumatology Expert to Lead Scleroderma Program at Cedars-Sinai’s Kao Institute

Francesco Boin, a nationally recognized rheumatologist, has taken over as director of the division of Rheumatology and of the new Scleroderma Program at Cedars-Sinai‘s ... Read more
June 15, 2020June 15, 2020
Shear-wave Elastography May Best Capture Changes in Skin Stiffness

A form of ultrasound imaging called shear-wave elastography (SWE) is more sensitive for assessing changes in skin stiffness over time in scleroderma (SSc) patients ... Read more
June 9, 2020June 10, 2020
Coalition Will Address Racial Disparities in Rare Disease Communities

The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting ... Read more

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March 16, 2018March 16, 2018

iBio Selects Fusion Protein as Lead Candidate for New Scleroderma Treatment

News

iBio has selected a lead scleroderma treatment candidate — a molecule it calls the E4-Fc fusion protein. The company plans to continue developing it, with a goal of moving it in clinical trials in people. It also hopes ... Read more

Updates

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

Rheumatology Expert to Lead Scleroderma Program at Cedars-Sinai’s Kao Institute

Shear-wave Elastography May Best Capture Changes in Skin Stiffness

Coalition Will Address Racial Disparities in Rare Disease Communities

TLY012 Receives FDA Orphan Drug Status for Systemic Scleroderma

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Experts Issue Guidance for Care of Scleroderma Patients During COVID-19

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Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

Lenabasum Use Seen to Lessen Inflammation, Fibrosis in Phase 2 dcSSc Trial

Early Use of Triple Combo Seen to Treat Diffuse Cutaneous Scleroderma

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

Ofev Approved in Europe for SSc-ILD Treatment in Adults

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

Blocking CCR2 Protein May Ease Scleroderma Symptoms, Mouse Study Suggests

Horizon Plans to Advance Oral dcSSc Therapy in Trials, Having Acquired Curzion

NORD’s Advice on COVID-19: You’re Stronger Than You Think

Phase 2 Trial to Test Cannabidiol-derived EHP-101 in Diffuse Cutaneous Scleroderma

Canada Clears EHP-101, Possible Cannabidiol-derived Treatment

NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum

Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

Subset of Immune B-Cells Linked to Vascular Damage in Some Patients, Study Suggests

Vitamin E, Pentoxifylline and TheraBite System Ease Lockjaw, Small Study Suggests

Emerald Health Awarded 6 Patents for Potential Cannabis Treatments

Blood Levels of Fibrotic Protein Tied to SSc-ILD, May Serve as Biomarker

Heart, Kidney Imaging Could Detect Fibrosis in Scleroderma, Study Finds

New Ultrasound Technique Detects Early Skin Changes in Scleroderma, Study Suggests

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

Tracleer Seen to Ease Skin Symptoms in Small Scleroderma Study

Cannabidiol Therapy, EHP-101, Put on Fast Track by FDA

iBio Selects Fusion Protein as Lead Candidate for New Scleroderma Treatment

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CellCept, Cyclophosphamide Both Improve Patient-reported Outcomes in SSc-ILD, Phase 2 Data Show

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

Rheumatology Expert to Lead Scleroderma Program at Cedars-Sinai’s Kao Institute

Shear-wave Elastography May Best Capture Changes in Skin Stiffness

Coalition Will Address Racial Disparities in Rare Disease Communities

Virtual Walks, Other Activities to Spotlight Scleroderma Awareness Month

TLY012 Receives FDA Orphan Drug Status for Systemic Scleroderma

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Experts Issue Guidance for Care of Scleroderma Patients During COVID-19

Subscribe to our mailing list

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

Lenabasum Use Seen to Lessen Inflammation, Fibrosis in Phase 2 dcSSc Trial

Early Use of Triple Combo Seen to Treat Diffuse Cutaneous Scleroderma

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

Ofev Approved in Europe for SSc-ILD Treatment in Adults

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

Blocking CCR2 Protein May Ease Scleroderma Symptoms, Mouse Study Suggests

Horizon Plans to Advance Oral dcSSc Therapy in Trials, Having Acquired Curzion

NORD’s Advice on COVID-19: You’re Stronger Than You Think

Phase 2 Trial to Test Cannabidiol-derived EHP-101 in Diffuse Cutaneous Scleroderma

Canada Clears EHP-101, Possible Cannabidiol-derived Treatment

NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum

Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

Subset of Immune B-Cells Linked to Vascular Damage in Some Patients, Study Suggests

Vitamin E, Pentoxifylline and TheraBite System Ease Lockjaw, Small Study Suggests

Emerald Health Awarded 6 Patents for Potential Cannabis Treatments

Blood Levels of Fibrotic Protein Tied to SSc-ILD, May Serve as Biomarker

Heart, Kidney Imaging Could Detect Fibrosis in Scleroderma, Study Finds

New Ultrasound Technique Detects Early Skin Changes in Scleroderma, Study Suggests

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

Tracleer Seen to Ease Skin Symptoms in Small Scleroderma Study

Cannabidiol Therapy, EHP-101, Put on Fast Track by FDA

Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare

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