News

Register Now for Global Genes’ RARE Patient Advocacy Summit

Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending the event in person in San Diego. “Here you’ll have the opportunity to connect and engage with others…

Uptravi IV Offers Important Option for Patients, Exec Says

In late July, the U.S. Food and Drug Administration (FDA) approved a new formulation of Janssen‘s Uptravi (selexipag) for use by people with pulmonary arterial hypertension (PAH). Uptravi already was available as an oral tablet. The new formulation is intravenous (IV) — meaning it is administered by an…

Peptide May Prevent PAH, Rat Study Finds

A peptide — a short chain of amino acids, the building blocks of proteins — called BPC 157 may prevent or counteract pulmonary arterial hypertension (PAH) and its complications, according to a preclinical study in a rat model of the disease. The findings suggest that BPC 157 may…

NORD Rare Disease Summit, Online Oct. 18-19, Open for Registration

Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also known as the National Organization for Rare Disorders (NORD) Summit, brings the rare disease community together to network and discuss developments in treatments and research…

Advocates Lobby US Congress During Virtual Rare Disease Week

More than 600 people participated in the 10th annual Rare Disease Week on Capitol Hill 2021, held virtually July 14–22, to advocate for the rare disease community. Hosted by the EveryLife Foundation’s Rare Disease Legislative Advocates (RDLA) program, the event brings together community members from across the U.S. to…

Pulnovo Raises Millions to Develop Its PAH Device and Trials

Pulnovo Medical recently raised an undisclosed amount totaling millions of dollars in investment funding to support clinical trials and product development related to pulmonary arterial hypertension (PAH). “The road of global medical innovation has never been an easy journey,” Cynthia Chen, Pulnovo’s executive president, said in a…

FDA Approves IV Form of Uptravi as Short-term Option to Oral Therapy

The U.S. Food and Drug Administration (FDA) has approved an intravenous form of Uptravi (selexipag) — one infused directly into the bloodstream — for people with pulmonary arterial hypertension (PAH) temporarily unable to take oral medicines. The new intravenous, or IV, formulation is intended to prevent treatment interruptions in adults…

PulmoSIM Planning to Bring PAH Therapy, PT001, Into Clinical Testing

PulmoSIM Therapeutics has joined with scientists at National Jewish Health hospital and Brown University to advance PT001, its investigational and potentially disease-modifying therapy for pulmonary arterial hypertension (PAH), into clinical trials. The partnership follows PT001 being designated an orphan drug by the U.S. Food and Drug Administration (FDA). Orphan drug status…

US Patient Groups Give Thumbs-Up to Rule Against Surprise Billing

The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a rule to protect consumers from surprise medical billing, in a joint statement with 26 other U.S. patient organizations. The interim final rule will implement patient protections required by the No Surprises Act. Surprise…