Updates

May 31, 2020June 1, 2020
TLY012 Receives FDA Orphan Drug Status for Systemic Scleroderma

The U.S. Food and Drug Administration has granted orphan drug designation to Theraly Fibrosis’ TLY012 for the treatment of systemic scleroderma. This ... Read more
May 28, 2020May 28, 2020
COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays ... Read more
May 25, 2020May 25, 2020
Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 ... Read more
May 20, 2020
Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together ... Read more
May 19, 2020May 19, 2020
Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together ... 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

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

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

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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

CHMP Favors EU Approval of Ofev to Preserve Lung Function in SSc-ILD

Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29

Panel Develops SSc-ILD Diagnosis, Management Guidelines

Inhibiting Key Protein May Ease Lung Scarring in Systemic Scleroderma, Study Suggests

Advice on Starting Nonprofit Groups for Rare and Other Diseases Focus of NORD Webinar

iBio Selects Fusion Protein as Lead Candidate for New Scleroderma Treatment

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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

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

Subscribe to our mailing list

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

CHMP Favors EU Approval of Ofev to Preserve Lung Function in SSc-ILD

Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29

Panel Develops SSc-ILD Diagnosis, Management Guidelines

Inhibiting Key Protein May Ease Lung Scarring in Systemic Scleroderma, Study Suggests

Advice on Starting Nonprofit Groups for Rare and Other Diseases Focus of NORD Webinar

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