Updates

August 12, 2020August 12, 2020
New Streaming Channel Showcases Rare Disease Films

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a ... Read more
August 10, 2020August 10, 2020
FLI1 Gene Variant Linked to Increased Scleroderma Risk, Greater Skin Thickness in Study

Long stretches of DNA repeats in the FLI1 gene — which gives instructions for a protein that controls the activity of genes — are ... Read more
August 3, 2020August 3, 2020
1st Patients Dosed, Enrollment Open in Phase 2a Trial of CBD Therapy EHP-101 for dcSSc

The first patients have been dosed in a Phase 2a clinical trial of EHP-101, Emerald Health Pharmaceuticals‘ investigational cannabidiol-derived treatment for diffuse cutaneous ... Read more
July 27, 2020July 27, 2020
Cell Therapy FCR001 to Enter Phase 1/2a Trial for Diffuse Cutaneous SSc

Talaris Therapeutics plans to initiate a Phase 1/2a trial to test its investigational cell therapy, FCR001, in people with diffuse cutaneous systemic sclerosis (dcSSc), ... Read more
July 20, 2020July 20, 2020
Sleep Apnea Linked to High Blood Pressure in Lungs of Adults With Scleroderma

Obstructive sleep apnea (OSA) is associated with an increased risk of pulmonary hypertension in people with scleroderma, a study suggests. The study, “Impact of concomitant obstructive ... 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 to ... Read more

Updates

New Streaming Channel Showcases Rare Disease Films

FLI1 Gene Variant Linked to Increased Scleroderma Risk, Greater Skin Thickness in Study

1st Patients Dosed, Enrollment Open in Phase 2a Trial of CBD Therapy EHP-101 for dcSSc

Cell Therapy FCR001 to Enter Phase 1/2a Trial for Diffuse Cutaneous SSc

Sleep Apnea Linked to High Blood Pressure in Lungs of Adults With Scleroderma

ACR CRISS Score a Reliable Measure of Therapy Effectiveness in dcSSc Patients, Corbus Says

Orencia Shows Promise in Treating Localized Scleroderma in Children, Adults

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

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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

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

iBio Selects Fusion Protein as Lead Candidate for New Scleroderma Treatment

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New Streaming Channel Showcases Rare Disease Films

FLI1 Gene Variant Linked to Increased Scleroderma Risk, Greater Skin Thickness in Study

1st Patients Dosed, Enrollment Open in Phase 2a Trial of CBD Therapy EHP-101 for dcSSc

Cell Therapy FCR001 to Enter Phase 1/2a Trial for Diffuse Cutaneous SSc

Sleep Apnea Linked to High Blood Pressure in Lungs of Adults With Scleroderma

ACR CRISS Score a Reliable Measure of Therapy Effectiveness in dcSSc Patients, Corbus Says

National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US

Orencia Shows Promise in Treating Localized Scleroderma in Children, Adults

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

Subscribe to our mailing list

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

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

Pin It on Pinterest