Updates

August 15, 2019August 13, 2019
Peripheral Neuropathy Alternative Treatments

Caregivers of patients suffering from symptoms of familial amyloid polyneuropathy are familiar with the prescribed treatments for the disease. Medications such as Neurontin (gabapentin) ... Read more
August 13, 2019August 13, 2019
Tegsedi and Onpattro Effectively Stop FAP Progression, Improve Quality of Life, Review Suggests

Tegsedi (inotersen) and Onpattro (patisiran) are both effective treatments to stop the progression of familial amyloid polyneuropathy (FAP), alleviate gastrointestinal symptoms, and improve patients’ overall ... Read more
August 8, 2019August 6, 2019
Managing Stress and Anxiety in Neuropathy Patients

The stresses of everyday life weigh heavily on patients with familial amyloid polyneuropathy. When faced with major external stressors, such as the diagnosis of ... Read more
August 6, 2019August 6, 2019
Ala97Ser Most Common Mutation Among Chinese-Malaysians With FAP, Study Says

The Ala97Ser mutation is the most common variant found on the TTR gene that has been associated with familial amyloid polyneuropathy (FAP) in Chinese-Malaysian ... Read more
August 1, 2019
Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate

Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in ... Read more

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News

Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card
August 1, 2019
Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of ...
Columns, Rumination and Response - a Column by Ezekiel Lim

Caregivers, Polyneuropathy, and Sun Exposure
August 1, 2019July 30, 2019
Summer months can be difficult for patients with familial amyloid polyneuropathy. Peripheral neuropathy symptoms ...
News

Early FAP Diagnosis Possible with Specific Sensory, Heart Rate and Conductance Tests, Study Says
July 30, 2019July 30, 2019
Measuring thresholds of cold detection, heart rate variability, and the skin’s electrical conductance may ...
Columns, Rumination and Response - a Column by Ezekiel Lim

What Caregivers Should Know About Peripheral Neuropathy Foot Treatments
July 25, 2019July 23, 2019
During the years that I’ve been acquainted with my mother-in-law, I’ve familiarized myself with ...
News

hATTR Compass, Free Genetic Testing and Counseling Program, Expanding
July 23, 2019July 23, 2019
Ambry Genetics and Akcea Therapeutics, an affiliate of Ionis Pharmaceuticals, are expanding a free ...
News

European Initiative Targets Diagnosis, Treatment of Rare Diseases
July 19, 2019
A new international consortium based in Paris, and funded largely by the 28-member European ...

Caregivers Should Be Mindful of Gabapentin’s Interactions with Other Medications
July 18, 2019July 17, 2019
For those suffering from symptoms of familial amyloid polyneuropathy — such as loss of ...
Risk of FAP Prompts Most Pre-symptomatic Neurological Testing, Portuguese Study Says
July 17, 2019July 17, 2019
Persons at risk for familial amyloid polyneuropathy (FAP) represent the vast majority of those ...
Caregivers Should Be Aware of Gabapentin’s Side Effects
July 11, 2019July 9, 2019
Long-term Efficacy of Vyndaqel in FAP Patients with Val30Met Mutations Supported in Study
July 9, 2019July 16, 2019
World’s First Alport Stamp Is Macedonian Mom’s Latest Win for Rare Disease Patients
July 4, 2019
Blood Biomarkers Show Inflammation in FAP Patients Before Amyloid Deposits Evident, Study Find
July 2, 2019July 2, 2019

Diet and Nutrition Help Relieve Peripheral Neuropathy Symptoms
June 27, 2019June 26, 2019
Familial amyloid polyneuropathy comes with plenty of symptoms that patients and caregivers must be ...
NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala
June 26, 2019June 26, 2019
Traveling with Nerve Pain Symptoms
June 20, 2019June 18, 2019
Eurordis Unveils Integrated-care Initiative for Rare Disease Patients
June 20, 2019

‘Rare Barometer’ Program Helps Eurordis Shape EU Rare Disease Policy
June 18, 2019
People with rare diseases know that the right government policies can make a big ...
Late-onset FAP Associated with Misdiagnosis, Greater Severity, Brazilian Study Finds
June 11, 2019June 11, 2019
Variants in C1QA and C1QC Genes Associated with Age at Onset in Portuguese Families
June 4, 2019June 4, 2019

News

Agency Unveils RaDaR to Help Patient Groups Develop Rare Disease Registries
May 28, 2019May 28, 2019
RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims ...
Columns, Rumination and Response - a Column by Ezekiel Lim

Here’s How Caregivers Can Adapt to the Acceptance Stage of Grief
May 23, 2019May 21, 2019
The diagnosis of a rare disease in a loved one is enough to usher ...
News

#AANAM – Tegsedi Continues to Benefit FAP Patients for up to Two Years
May 21, 2019May 21, 2019
Tegsedi (inotersen) continues to slow progression of familial amyloid polyneuropathy (FAP) and maintains the ...
Columns, Rumination and Response - a Column by Ezekiel Lim

New Caregivers Should Be Proactive When Dealing with Depression Risk
May 16, 2019May 14, 2019
For new caregivers, depression is a clear and present danger. As the fourth of ...
News

Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns
May 15, 2019
With 250 rare diseases newly identified every year, scientists can barely keep up — ...
Columns, Rumination and Response - a Column by Ezekiel Lim

Addressing The Bargaining Stage of Grief as a Caregiver
May 2, 2019April 30, 2019
The bargaining stage of grief begins when the reality of a traumatic event sinks ...

September 6, 2017September 6, 2017

Tafamidis Delays Disease Progression in Patients with Transthyretin Amyloid Polyneuropathy, Pfizer Study Suggests

News

An interim analysis of the Phase 3 trial for tafamidis (Fx-1006A) as a therapy for patients with hereditary transthyretin amyloid polyneuropathy (TTR-FAP) shows the investigational treatment delays disease progression, Pfizer announced. The study, “Long-term safety and efficacy of tafamidis for ... Read more

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