GPP Features

Features, GPP Features

Lack of Diagnostic Consensus for GPP Poses Major Challenges for Physicians and Patients

By

Kyle Habet, MD

Publish Date August 24, 2023

Despite its distinctive clinical presentation, diagnosis of generalized pustular psoriasis (GPP) poses significant challenges for patients and physicians.

Features, GPP Features

Recognizing and Managing Flares in Generalized Pustular Psoriasis

By

Tori Rodriguez, MA, LPC, AHC

Publish Date July 26, 2023

Due to the rarity of generalized pustular psoriasis, cases often go unrecognized by physicians who are not dermatologists.

ATTR-CM Features, FA Features, Features, GPP Features, HD Features, LAL-D Features, MDS Features, PWS Features, SSc Features

Advocates Pushing for Safer, More Accessible Air Travel for People With Disabilities

By

Larry Luxner

Publish Date May 2, 2023

Rare disease advocacy groups are lobbying Congress to amend regulations to let people with disabilities remain in their wheelchairs during flights.

AATD Features, CF Features, Features, GPP Features, LAL-D Features, LGS Features, MDS Features, MG Features, Pompe Features, PWS Features, SSc Features

As Pandemic Restrictions Continue to Ease, Rare Disease Patients Still Fear COVID-19

By

Larry Luxner

Publish Date April 12, 2023

Three years after COVID-19 was declared a pandemic, rare disease patients still fear the contagion—and must deal with the fallout.

ANCA Features, ATTR-CM Features, CAD Features, CCA Features, CF Features, Features, GPP Features, HAE Features, HEM Features, IPF Features, LAL-D Features, LCFAOD Features, LGS Features, MDS Features, PNH Features, Pompe Features, SCD Features, SSc Features

At 40, Orphan Drug Act Enjoys Rare Bipartisan Support

By

Larry Luxner

Publish Date January 18, 2023

Rare disease advocates are marking the 40th anniversary of the landmark Orphan Drug Act, which has led to over 1100 approvals for new therapies.

ALGS Features, ATTR-CM Features, DLBCL Features, DMD Features, Features, GPP Features, HEM Features, MDS Features, MG Features, NMOSD Features, PAH Features, PNH Features, Pompe Features, SM Features

Ambitious NCATS Bespoke Gene Therapy Consortium Targets Ultrarare Diseases

By

Larry Luxner

Publish Date December 29, 2022

The Bespoke Gene Therapy Consortium (BGTC) is an ambitious public-private partnership that aims to develop gene therapies for ultrarare diseases.

ALGS Features, ANCA Features, Features, GPP Features, LAL-D Features, LAL-D News Briefs, LGS Features, MDS Features, News Briefs, Pompe Features, WD Features

EveryLife Foundation Navigates Complex Issues to Advocate for Rare Disease Patients

By

Larry Luxner

Publish Date November 21, 2022

The EveryLife Foundation for Rare Diseases pushes a legislative agenda aimed at benefitting the 30 million Americans with a rare disease.

AATD Features, CF Features, DMD Features, Features, GPP Features, HEM Features, IPF Features, LAL-D Features, LCFAOD Features, MG Features, MS Features, MTC Features, SCD Features, SMA Features

The Alliance for Regenerative Medicine Fights for Better Cell, Gene Therapies

By

Larry Luxner

Publish Date August 25, 2022

The Alliance for Regenerative Medicine aims to help in the development of safe and effective regenerative therapies that can benefit rare disease patients.

Features, GPP Features, LCFAOD Features

‘Fundamental Flaws’ Plague Drug Development Process, Ultragenyx CEO Dr. Emil Kakkis Says

By

Larry Luxner

Publish Date August 2, 2022

Ultragenyx CEO Dr. Emil D. Kakkis said federal regulators should focus more on biomarkers and less on clinical endpoints when it comes to drug approvals.

CCA Features, CF Features, Features, GPP Features, NMOSD Features, PAH Features, SM Features

An Inspirational Feat: Crossing Spain’s 500-Mile Camino de Santiago by Wheelchair

By

Larry Luxner

Publish Date July 11, 2022

When 2 friends set out along Spain’s 500-mile Camino de Santiago, they didn’t know how many people they’d inspire with 3 little words: “I’ll Push You.”