Updates

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 27, 2020May 27, 2020
Clinical Trials and AADC Deficiency

There have been only about 100 cases of aromatic L-amino acid decarboxylase (AADC) deficiency described in the medical literature. It is very difficult to ... Read more
May 26, 2020May 26, 2020
AADC Patients Experience Severe Symptoms from Infancy to Adulthood, Study Reveals

Most patients with aromatic l-amino acid decarboxylase (AADC) deficiency experience a combination of severe motor symptoms and prominent non-motor symptoms that persist from childhood to adulthood, according ... 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, 2020May 20, 2020
Anesthesia and AADC Deficiency

Medical procedures and tests sometimes require anesthesia, which can be especially challenging for patients with aromatic L-amino acid decarboxylase (AADC) deficiency, a rare genetic ... Read more

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Updates

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

Clinical Trials and AADC Deficiency

AADC Patients Experience Severe Symptoms from Infancy to Adulthood, Study Reveals

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Anesthesia and AADC Deficiency

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

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

What’s a Dystonic Crisis with AADC Deficiency, and How to Deal With It?

PTC Therapeutics to Seek FDA Approval of AADC Gene Therapy

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

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

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Medications to Avoid in AADC Deficiency

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

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

AADC Activity Can Be Assessed with High Resolution FMT-PET, Study Shows

What Equipment Will my Child with AADC Deficiency Need?

New Method May Help Diagnose AADC Deficiency, Other Genetic Disorders of Metabolism

Mindfulness for Those Caring for a Patient With AADC Deficiency

Spoon Theory for AADC Deficiency Caregivers

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

Importance of Support Networks for People Affected by AADC Deficiency

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

AADC Deficiency May Be ‘Common’ in Neurological Problems of Unknown Origin

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

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

Sleeping Tips for Children With AADC Deficiency

Patients, Supporters Worldwide Recognizing Rare Disease Day 2020

2 New DDC Gene Mutations, Identified in African American Siblings, Described in Case Report

Aquatic Therapy for Children with AADC Deficiency

FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar

With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

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

Clinical Trials and AADC Deficiency

AADC Patients Experience Severe Symptoms from Infancy to Adulthood, Study Reveals

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Anesthesia and AADC Deficiency

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

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

What’s a Dystonic Crisis with AADC Deficiency, and How to Deal With It?

PTC Therapeutics to Seek FDA Approval of AADC Gene Therapy

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

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

Subscribe to our mailing list

Medications to Avoid in AADC Deficiency

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

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

AADC Activity Can Be Assessed with High Resolution FMT-PET, Study Shows

What Equipment Will my Child with AADC Deficiency Need?

New Method May Help Diagnose AADC Deficiency, Other Genetic Disorders of Metabolism

Mindfulness for Those Caring for a Patient With AADC Deficiency

Spoon Theory for AADC Deficiency Caregivers

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

Importance of Support Networks for People Affected by AADC Deficiency

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

AADC Deficiency May Be ‘Common’ in Neurological Problems of Unknown Origin

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

Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare

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

Sleeping Tips for Children With AADC Deficiency

Patients, Supporters Worldwide Recognizing Rare Disease Day 2020

2 New DDC Gene Mutations, Identified in African American Siblings, Described in Case Report

Aquatic Therapy for Children with AADC Deficiency

FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar

With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

Pin It on Pinterest