Meagan Fulps, Author at Patient Worthy

Source: Pixabay

Cycle Pharmaceuticals Partners With US-Firm To Utilize 3D Printing Technology For Rare Disease Treatment Development

Post author:Meagan Fulps
Post published:January 10, 2018
Post category:Rare Disease Tyrosinemia

Cycle Pharmaceuticals shook hands with US-based firm, Aprecia Pharmaceuticals as they agreed to develop and commercialize treatments for rare diseases using the advanced technology of 3D printing, recently reported Cambridge…

Source: Pixabay

Gene Therapy Makes Noise in 2017

Post author:Meagan Fulps
Post published:January 10, 2018
Post category:Cutaneous T-cell Lymphomas Rare Disease RPE65-associated Leber Congenital Amaurosis

Gene therapy has successfully made its presence known in the medical field this last year, reported ABC News. Dr. Francis Collins of the National Institutes of Health isn't afraid to…

Source: Pixabay

Anna Doesn’t Let Friedreich Ataxia Define Her

Post author:Meagan Fulps
Post published:January 9, 2018
Post category:Friedreich ataxia Rare Disease

Anna Gordon from West Virginia doesn't let her Friedreich ataxia define her, and she encourages anyone with a rare disease to not let it define them either, reported Public Broadcasting.…

Source: Pixabay

U.K. Gets Approval For $700,000 SCID Gene Therapy Treatment

Post author:Meagan Fulps
Post published:January 8, 2018
Post category:Primary Immunodeficiencies SCID

'Tis the season for gene therapy approvals. Reported by Bloomberg, the U.K. just approved its most expensive medication to date. The treatment is for severe combine immune deficiency, or more…

Source: Pixabay

Gene Therapy Luxturna Will Cost $850,000 To Patients With Rare Eye Disease

Post author:Meagan Fulps
Post published:January 8, 2018
Post category:Rare Disease RPE65-associated Leber Congenital Amaurosis

The FDA passed along their nod of approval for the sought after gene therapy treatment, Luxturna, reported Engadget. This will be used to help aid those made blind by a…

Source: Pixabay

Supermom Cori Salchert Fosters Terminally Ill Children

Post author:Meagan Fulps
Post published:January 8, 2018
Post category:Hypoxic Ischemic Encephalopathy Rare Disease

Cori Salchert is a hero. Recently featured on the Today Show, Cori was honored for being the ultimate supermom, taking in foster children who are sick with terminal diseases, report…

Source: Pixabay

A Boy With Batten Disease Touches America With Viral Video Listening To An Ed Sheeran Song

Post author:Meagan Fulps
Post published:January 5, 2018
Post category:Batten Disease

Music is sometimes more powerful than we even realize on the daily. Music keeps little 6-year-old Ollie Carroll going, even though he understands his life will be short. Ollie and…

Source: Pixabay

Strong Parents Care for Their Two Young Children with Rare Conditions

Post author:Meagan Fulps
Post published:January 5, 2018
Post category:Hereditary hemorrhagic telangiectasia Rare Disease

No family faces life's unexpected challenges better than the family of Bella and Liam Brya. Allison and her husband, Bill, deal with the struggles of having two children, Bella and…

Source: Pixabay

Aspiring Physician Assistant with Keratoconus Avoids Blindness with Experimental Cross-Linking Procedure

Post author:Meagan Fulps
Post published:January 5, 2018
Post category:Keratoconus Rare Disease

In 2009 Emily Rochac Argueta realized her vision was rapidly deteriorating headed towards blindness, reported Times Union. The aspiring physician assistant was diagnosed with keratoconus, a rare eye disease that…

Source: Pixabay

35th Anniversary for NORD and The Orphan Drug Act

Post author:Meagan Fulps
Post published:January 5, 2018
Post category:Rare Disease

It's been 35-years since the passing of the Orphan Drug Act, and creation of the National Organization for Rare Disorders (NORD), reports PR Newswire. Even though these organizations recognize there…

Source: Pixabay

High School Student with Isovaleric Acidemia Beats the Odds and Plays Football

Post author:Meagan Fulps
Post published:January 4, 2018
Post category:isovaleric acidemia Rare Disease

High school football player McCaulley McGuire doesn't let his isovaleric acidemia keep him from playing the game, reported Times Free Press. Diagnosed with the rare disorder when he was only…

Source: Pixabay

Wife Stays Positive After Death of Husband Fighting Amyotrophic Lateral Sclerosis

Post author:Meagan Fulps
Post published:January 4, 2018
Post category:Amyotrophic Lateral Sclerosis Rare Disease

Nanette Whitfield, stood beside her best friend and husband as amyotrophic lateral sclerosis (ALS) painfully took his life, reported The Record Courier. Watching someone you love slowly lose everything has…

https://pixabay.com/en/baby-girl-sleep-child-toddler-1151351/

Mother of Child with NKH Writes Children’s Book To Fund Research

Post author:Meagan Fulps
Post published:January 4, 2018
Post category:nonketotic hyperglycinemia Rare Disease

It's hard to find funding for rare diseases research. Patients and the people who love them don't have the same established networks of support that help people struggling with more…

https://pixabay.com/en/people-woman-travel-adventure-trek-2591874/

Loin Pain Hematuria Syndrome Sidelines an Active 28-Year-Old

Post author:Meagan Fulps
Post published:January 3, 2018
Post category:Loin Pain Hematuria Syndrome

The active and adventurous Devaney Mehl was sidelined by excruciating pain when only 28-years-old, originally reported in Regina Leader-Post. After many incorrect diagnoses, she later learned she had developed the rare…

Source: Pixabay

Screening Test for Navajo Newborns Saves Babies Suffering from Severe Combined Immunodeficiency (SCID)

Post author:Meagan Fulps
Post published:January 3, 2018
Post category:Primary Immunodeficiencies SCID

Joy Littlesunday is just one of many Navajo newborns who were screened and thus saved from severe combined immunodeficiency by a successful bone marrow transplant at UC San Francisco, reported UCSF…

Source: Pixabay

Chinese Family Faces Medical Financial Hardships Trying to Keep Their Son Alive with Hypoxic Ischemic Encephalopathy

Post author:Meagan Fulps
Post published:January 3, 2018
Post category:Hypoxic Ischemic Encephalopathy Rare Disease

The Chinese Zhiying family face their ultimate breaking point when they learn they can no longer afford the medical bills needed to save their 7-month-old son, Yiwei, who is fighting…

Source: Pixabay

8-Year-Old Boy Selected to Meet His Idols of Passed Boston Bruin’s Hockey Players

Post author:Meagan Fulps
Post published:January 2, 2018
Post category:Hereditary hemorrhagic telangiectasia

Charlie Druken's dreams came true when he was selected to the drop the puck at the Boston Bruins' Alumni game recently, reported The Telegram. It was a night the eight-year-old…

Source: Pixabay

11-Year-Old Receives Life Saving Kidney For Christmas

Post author:Meagan Fulps
Post published:January 2, 2018
Post category:Focal segmental glomerulosclerosis

It all started with a headache. 11-year-old Canyon Martin wasn't acting like himself and was sent home from school with a headache, reported Standard Times. A quick doctor visit after…

Source: Pixabay

Family Continues the Search for a Liver Donor for Their Baby

Post author:Meagan Fulps
Post published:January 1, 2018
Post category:Biliary Atresia

Ryan Cole is a beautiful baby. You would never know from looking at him that he was struggling for life fighting a rare disease called biliary atresia, reported Courier Mail.…

Source: Pixabay

Video of Exuberant Man Meeting Santa Claus Goes Viral

Post author:Meagan Fulps
Post published:December 29, 2017
Post category:15Q24

When 24-year-old Andy Pallone finally met Santa Claus, it heart-warmed his mother, and unexpectedly the rest of America, reported Belleville News-Democrat. His mother, Jennifer Pallone, wanted to capture the moment…

Source: Pixabay

Woman Remains Blessed Amidst Idiopathic Intracranial Hypertension Diagnosis

Post author:Meagan Fulps
Post published:December 29, 2017
Post category:Intracranial Hypertension Rare Disease

While April Archote faces the physical pain and financial hardships of living with a rare brain disease, she continues to be positive and count her blessings, CCHeadliner reported. In 2013, April was…

Source: Pixabay

NORD Sends Happy Holiday Wishes and Update

Post author:Meagan Fulps
Post published:December 29, 2017
Post category:Rare Disease

NORD, the National Organization for Rare Diseases, sent out their happiest wishes for the holidays, their utmost gratitude for 2017, as well as their plan of attack for the New…

Source: Pixabay

Progress Has Been Made For Orphan Drug Research, But Is It Enough?

Post author:Meagan Fulps
Post published:December 28, 2017
Post category:Rare Disease

According to The Pharma Letter, over the last 20 years progress with the development of orphan drugs for rare diseases has been made, but many question, is it enough? There…

Source: Pixabay

Family pays $24,000 monthly to keep their child living with Gaucher disease alive

Post author:Meagan Fulps
Post published:December 28, 2017
Post category:Gaucher Disease Rare Disease

Originally reported by The Straits Times, the Chew family from Singapore does everything they can to keep their nine-year-old daughter alive, even if it means spending their life savings and…

Source: Pixabay

7-Year-Old with Charcot-Marie-Tooth Nears Fundraising Goal That Would Let Him Walk Again

Post author:Meagan Fulps
Post published:December 27, 2017
Post category:Charcot-Marie-Tooth disease

Just like any other 7-year-old little boy, Ethan Perkins has big dreams. He loves trucks and reading, and wants to study fossils as a paleontologist reported KXLY. He also wants to be…

Cycle Pharmaceuticals Partners With US-Firm To Utilize 3D Printing Technology For Rare Disease Treatment Development

Gene Therapy Makes Noise in 2017

Anna Doesn’t Let Friedreich Ataxia Define Her

U.K. Gets Approval For $700,000 SCID Gene Therapy Treatment

Gene Therapy Luxturna Will Cost $850,000 To Patients With Rare Eye Disease

Supermom Cori Salchert Fosters Terminally Ill Children

A Boy With Batten Disease Touches America With Viral Video Listening To An Ed Sheeran Song

Strong Parents Care for Their Two Young Children with Rare Conditions

Aspiring Physician Assistant with Keratoconus Avoids Blindness with Experimental Cross-Linking Procedure

35th Anniversary for NORD and The Orphan Drug Act

High School Student with Isovaleric Acidemia Beats the Odds and Plays Football

Wife Stays Positive After Death of Husband Fighting Amyotrophic Lateral Sclerosis

Mother of Child with NKH Writes Children’s Book To Fund Research

Loin Pain Hematuria Syndrome Sidelines an Active 28-Year-Old

Screening Test for Navajo Newborns Saves Babies Suffering from Severe Combined Immunodeficiency (SCID)

Chinese Family Faces Medical Financial Hardships Trying to Keep Their Son Alive with Hypoxic Ischemic Encephalopathy

8-Year-Old Boy Selected to Meet His Idols of Passed Boston Bruin’s Hockey Players

11-Year-Old Receives Life Saving Kidney For Christmas

Family Continues the Search for a Liver Donor for Their Baby

Video of Exuberant Man Meeting Santa Claus Goes Viral

Woman Remains Blessed Amidst Idiopathic Intracranial Hypertension Diagnosis

NORD Sends Happy Holiday Wishes and Update

Progress Has Been Made For Orphan Drug Research, But Is It Enough?

Family pays $24,000 monthly to keep their child living with Gaucher disease alive

7-Year-Old with Charcot-Marie-Tooth Nears Fundraising Goal That Would Let Him Walk Again

The Let’s Chat CAR T One-on-One Mentor Program: Speaking with Someone Who Understands What You Are Going Through

“Shades of Psoriasis”: Plaque Psoriasis and Sharing Your Story

Understanding RSV: Why Combatting Ethnic and Age-Related Health Disparities Could Improve Outcomes

We Need Equity and Diversity Within Rare Disease Research

DNA in Color: How Ronya Nelson is Working to Close the Diversity Gap in Genetics - Part 1

Bridging the Diversity Gap in Healthcare

Congratulations to the 2023 Recipients of the Diversity in Lupus Research Awards!

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